From High-Level Declaration to a Blueprint for Action: What the High-Level Meeting on rare diseases confirmed about Europe’s potential

From High-Level Declaration to a Blueprint for Action: What the High-Level Meeting on rare diseases confirmed about Europe’s potential December 2025 Europe’s rare disease community marked an important milestone last week as the three-day High-Level Meeting on European Research and Innovation for Rare Diseases concluded in Brussels. Across keynote speeches, panel discussions and political interventions, one message came through clearly: rare diseases require a coherent, long-term policy vision that no Member State can deliver alone. https://www.eurordis.org/from-high-level-declaration-to-blueprint-for-action-plan/

Parenting a Child with Smith-Lemli-Opitz Syndrome January 2026

Parenting a Child with Smith-Lemli-Opitz Syndrome January 2026 In this interview led by Matt Bolz-Johnson, our Former ATMP & Mental Health Advisor, Antje Enekwe reflects on raising a child with Smith-Lemli-Opitz syndrome. Through an unexpected diagnosis and the daily realities of parenting a child with Smith-Lemli-Opitz syndrome, Antje Enekwe shares how she navigated sleepless nights and major life changes, and found resilience through a support network – holding onto small moments of joy that continue to shape their family’s future. https://www.eurordis.org/parenting-a-child-with-smith-lemli-opitz-syndrome/

Rita Francisco: More Clinical Trial Innovation Than You Can Imagine to be RealiseD January 2026

Rita Francisco: More Clinical Trial Innovation Than You Can Imagine to be RealiseD January 2026 RealiseD is an Innovative Health Technology (IHI) project that aims to accelerate clinical trial innovation for rare and ultra-rare diseases. From participant recruitment to trial design, regulatory acceptance and clinical trial site standards, RealiseD is striving to make an impact on the clinical trial ecosystem. Will we succeed? https://www.eurordis.org/rita-francisco-clinical-trial-innovation-realised/

A message from Virginie Hivert, our Acting CEO, to our Members

A message from Virginie Hivert, our Acting CEO, to our Members January 2026 On behalf of EURORDIS – Rare Diseases Europe, I extend my very best wishes for the New Year to all of our Members and our wider community of rare disease advocates. https://www.eurordis.org/message-from-our-acting-ceo-to-our-members/

Building global consensus: Why unified terminology matters for childhood dementia and all rare diseases

Building global consensus: Why unified terminology matters for childhood dementia and all rare diseases January 2026 The Member Comment article below was shared with EURORDIS-Rare Diseases Europe by Kris Kusidlo, Chairman of our member organisation Sanfilippo Initiative, and Community Representative of the Global Childhood Dementia Consensus Project Steering Committee. https://www.eurordis.org/why-unified-terminology-matters-for-childhood-dementia/

ECRD 2026: Advancing rare disease policies in a changing and competitive Europe

ECRD 2026: Advancing rare disease policies in a changing and competitive Europe January 2026 The 13th European Conference on Rare Diseases and Orphan Products (ECRD 2026) will take place in Prague and online on 3 and 4 June under the theme “Rare Diseases in a Changing & Competitive Europe: Shaping policies to address the unmet needs of people living with rare diseases”. The conference will bring together a broad range of stakeholders to advance concrete policy solutions that improve the lives of people living with a rare disease and their families. https://www.eurordis.org/ecrd-2026-advancing-rare-disease-policies-in-a-changing-europe/

A Call for an EU Budget that Delivers for Health, Cancer, and Rare Diseases Tuesday, February 3, 2026 10:00 PM 11:30 PM

https://www.intergrouponcancerandrarediseases.eu/events/a-call-for-an-eu-budget-that-delivers-for-health-cancer-and-rare-diseases With negotiations ongoing on the EU’s next long-term budget for 2028–2034, MEPs from across political groups will come together on 3 February for a meeting of the European Parliament Intergroup on Cancer and Rare Diseases, titled ‘A Call for an EU Budget that Delivers for Health, Cancer, and Rare Diseases’. The Intergroup will warn that health risks are being sidelined in EU budgetary decisions and will issue a joint declaration calling for stronger and more explicit commitments to key EU initiatives on cancer and rare diseases in the next Multiannual Financial Framework (MFF) for 2028–2034. While significant progress has been made over the past two decades to improve the lives of people living with rare diseases through initiatives such as the European Reference Networks and ERDERA, the rare disease community is still waiting for a coherent EU framework comparable to Europe’s Beating Cancer Plan. As the next MFF will shape EU policy action for nearly a decade, the upcoming Intergroup meeting represents an opportunity to secure sustained funding and political commitment to support people living with cancer and rare diseases.

The Link Between Chronic Pain and Depression – In-Depth Doctor’s Interview By Ivanhoe Broadcast News on June 13, 2024

https://www.ivanhoe.com/interview/the-link-between-chronic-pain-and-depression-in-depth-doctors-interview/ Kurt Kroenke, MD, Research Scientist at Regenstrief Institute, talks about the link between chronic pain and depression. Interview conducted by Ivanhoe Broadcast News in 2024.

Ways To Grow Younger, Not Older By Ivanhoe Broadcast News on February 6, 2026

https://www.ivanhoe.com/?p=36626&preview=1&_ppp=b5e99fdade People who feel younger than they are not only have a more positive lookout on life but also better mental and physical health. Find out some easy ways to grow younger.

Options For Prostate Cancer By Ivanhoe Broadcast News on February 5, 2026

https://www.ivanhoe.com/?p=36623&preview=1&_ppp=296a74176f There are now more options than ever for treating prostate cancer. Learn what they are and what research is saying about them.

Inside The Brain: Transforming Parkinson’s By Ivanhoe Broadcast News on February 4, 2026

https://www.ivanhoe.com/?p=36620&preview=1&_ppp=024121e264 More than one million Americans are living with Parkinson’s. Find out what researchers are hoping is the next big breakthrough for treatment.

Plasma Power: Saving Brains After TBI By Ivanhoe Broadcast News on February 3, 2026

https://www.ivanhoe.com/?p=36616&preview=1&_ppp=4a3f4cbfda A head injury can change a person’s life forever and there has been no specific treatment for it. But now doctors have found something new that could change that.

Rod’s Remarkable Recovery from a Rare Diagnosis By Ivanhoe Broadcast News on February 2, 2026

https://www.ivanhoe.com/?p=36612&preview=1&_ppp=91b7b23392 About one in 10 Americans will be diagnosed with a rare disease and getting that diagnosis isn’t easy. Learn about one man who refused to give up on getting his life back

Ongoing Perspectives in HER2-Mutated Advanced NSCLC: An Expert Viewpoint on the Latest Data ++

CME/CE Courses: Ongoing Perspectives in HER2-Mutated Advanced NSCLC: An Expert Viewpoint on the Latest Data CME / ABIM MOC Ongoing Perspectives in HER2-Mutated Advanced NSCLC: An Expert Viewpoint on the Latest Data Authors: Helena Yu, MD https://www.medscape.org/viewarticle/ongoing-perspectives-her2-mutated-advanced-nsclc-expert-2026a10001va?page=1&sso=true&impID=8068641&uac=148436CN&src=wnl_tpal_260130_mscpedu HER2-Mutated NSCLC: Are Your Clinical Decisions on Target? CME / ABIM MOC HER2-Mutated NSCLC: Are Your Clinical Decisions on Target? Authors: Nicolas Girard, MD, PhD https://www.medscape.org/viewarticle/her2-mutated-nsclc-are-your-clinical-decisions-target-2026a10000gu?page=1&sso=true&impID=8068641&uac=148436CN&src=wnl_tpal_260130_mscpedu

lipping the Script: De-escalation Trials Need Superiority, Not Noninferiority ++++

News & Perspectives: Autism Experts Concerned Over Federal Advisory Panel Overhaul Autism Experts Concerned Over Federal Advisory Panel Overhaul Alicia Ault https://www.medscape.com/viewarticle/autism-experts-concerned-over-federal-advisory-panel-2026a10002xo?ecd=wnl_edit_tpal_etid8069481&uac=148436CN&impID=8069481 January 29, 2026 MASLD for Cardiologists The Basics of Metabolic Dysfunction–Associated Steatotic Liver Disease for Cardiologists Pathophysiology, Diagnosis, and Treatment Muhammad Shahzeb Khan, MD, MSC; Syed Sarmad Javaid, MBBS; Amreen Dinani, MD; Kara Wegermann, MD; Ambarish Pandey, MD; Ankeet S. Bhatt, MD, MBA, SCM; Mark Muthiah, MBBS; Harriette G.C. Van Spall, MD, MPH; Faiez Zannad, MD, PHD; Javed Butler, MD, MPH, MBA; Michael L. Volk, MD; Marat Fudim, MD, MHS https://www.medscape.com/s/viewarticle/1003217?ecd=wnl_edit_tpal_etid8069481&uac=148436CN&impID=8069481 J Am Coll Cardiol. 2025;86(20):1861–1884 Flipping the Script: De-escalation Trials Need Superiority, Not Noninferiority Flipping the Script: De-escalation Trials Need Superiority, Not Noninferiority Bishal Gyawali, MD, PhD https://www.medscape.com/viewarticle/flipping-script-de-escalation-trials-need-superiority-not-2026a1000220?ecd=wnl_edit_tpal_etid8069481&uac=148436CN&impID=8069481 January 29, 2026 Withdrawing From the WHO: America Is Not First in Isolation Withdrawing From the WHO: America Is Not First in Isolation Demetre C. Daskalakis, MD, MPH https://www.medscape.com/viewarticle/withdrawing-who-america-not-first-isolation-2026a10002ma?ecd=wnl_edit_tpal_etid8069481&uac=148436CN&impID=8069481 January 29, 2026

Time of radiotherapy can influence the effectiveness of cancer treatment

https://www.news-medical.net/news/20260130/Time-of-radiotherapy-can-influence-the-effectiveness-of-cancer-treatment.aspx A team of researchers from the Andalusian Centre for Molecular Biology and Regenerative Medicine (CABIMER) and the University of Seville, in collaboration with the Virgen Macarena University Hospital, have identified a fundamental mechanism that links the 24-hour circadian cycle to the precise repair of DNA breaks. This study, which focused on the circadian protein Cryptochrome1 (CRY1), suggests that the time of day when radiotherapy is administered can significantly influence the effectiveness of treatment for certain types of cancer.

Editorial Broadening applications for intrathecal gene therapy: a case for lysosomal storage diseases eBioMedicine +++ +...

Projected extreme temperature event-attributable dementia deaths in China: a climate–ageing–adaptation framework Rui Zhangb,c ∙ Yonghong Lia liyonghong@nieh.chinacdc.cn ∙ Huan Zhengb ∙ Mulei Chenb ∙ Jia Zhaob ∙ Yiming Huc ∙ et al. https://www.thelancet.com/journals/ebiom/article/PIIS2352-3964(25)00522-5/fulltext?dgcid=hubspot_update_feature_updatealerts_ebiom&utm_campaign=update-ebiom&utm_medium=email&_hsenc=p2ANqtz-9709_FH6DDpD6MWaQ4QMWGxBzU3u4XMy26EnYmtU7_92L5_ftsYMq5XOjmrz7Na4SyE9_yoihy7rG4ojTFxJZsE_zZhw&_hsmi=400872454&utm_content=400412095&utm_source=hs_email Prediction of brain age using structural magnetic resonance imaging: a comparison of clinical utility of publicly available software packages Ruben P. Dörfela,b,c ruben.dorfel@ki.se ∙ Brice Ozenneb,d ∙ Melanie Ganzb,c ∙ Jonas E. Svenssona ∙ Pontus Plavén-Sigraya,b for the Alzheimer's Disease Neuroimaging Initiative (ADNI) https://www.thelancet.com/journals/ebiom/article/PIIS2352-3964(25)00544-4/fulltext?dgcid=hubspot_update_feature_updatealerts_ebiom&utm_campaign=update-ebiom&utm_medium=email&_hsenc=p2ANqtz-9hC0a1NEg9JwizX6PYix9Ina8b0NAXbAav4bNDQM6A30a6edOlVW_IfNoCQdrLAPmcfyRy-KvNxd8Th2CZ3hKmDMe8Cw&_hsmi=400872454&utm_content=400412095&utm_source=hs_email Paediatric cerebrospinal fluid immune profiling distinguishes paediatric-onset multiple sclerosis from other paediatric-onset acute neurological disorders Diego A. Espinozaa,b,c,o ∙ Tobias Zrzavya,b,d,o ∙ Gautier Brevillea,b,o ∙ Simon Thebaulta,b,e ∙ Amaar Marefib,f,g ∙ Ina Mexhitaja,b ∙ et al. https://www.thelancet.com/journals/ebiom/article/PIIS2352-3964(25)00538-9/fulltext?dgcid=hubspot_update_feature_updatealerts_ebiom&utm_campaign=update-ebiom&utm_medium=email&_hsenc=p2ANqtz-_2zZLUnSNhYCkJAVS32d7UaED544qgt3AiSdYvzGARR6Z4yUQcErmCQ56bj-i959qwtDTDEbPI5twvJu-ZtSUjqQ-3vw&_hsmi=400872454&utm_content=400412095&utm_source=hs_email Editorial Broadening applications for intrathecal gene therapy: a case for lysosomal storage diseases eBioMedicine https://www.thelancet.com/journals/ebiom/issue/vol123nonull/PIIS2352-3964(25)X0012-8

Call for letters The Lancet Infectious Diseases ++... ++

Call for letters The Lancet Infectious Diseases https://www.thelancet.com/journals/laninf/article/PIIS1473-3099(26)00010-1/fulltext?dgcid=raven_jbs_etoc_email Feb 2026 Volume 26Number 2p111-212, e77-e131 https://www.thelancet.com/journals/laninf/issue/vol26no2/PIIS1473-3099(26)X2001-1 Long COVID associated with SARS-CoV-2 reinfection among children and adolescents in the omicron era (RECOVER-EHR): a retrospective cohort study Bingyu Zhang, MSa,b ∙ Qiong Wu, PhDa,c,d ∙ Prof Ravi Jhaveri, MDf ∙ Ting Zhou, MD PhDa,c ∙ Prof Michael J Becich, MD PhDe ∙ Yuriy Bisyuk, MD PhDg ∙ et al. https://www.thelancet.com/journals/laninf/article/PIIS1473-3099(25)00476-1/fulltext?dgcid=raven_jbs_etoc_feature_laninf Characterising household transmission dynamics of clade Ib mpox in Burundi: a prospective cohort study Raoul Kamadjeu, MDa,b rkamadjeu@unicef.org ∙ Ferdinand Nsengimana, PhDc ∙ Manassé Nimpagaritse, PhDc ∙ Edna Moturi, MDd ∙ Eric Kezakarayagwa, BScc ∙ Rose Nkiko, MDe ∙ et al. https://www.thelancet.com/journals/laninf/article/PIIS1473-3099(25)00483-9/abstract?dgcid=raven_jbs_etoc_feature_laninf

Resident doctors: an investment in the future of the NHS The Lancet ++ + +...

Interval cancer, sensitivity, and specificity comparing AI-supported mammography screening with standard double reading without AI in the MASAI study: a randomised, controlled, non-inferiority, single-blinded, population-based, screening-accuracy trial Jessie Gommers, MSca,b ∙ Veronica Hernström, MDb,c ∙ Viktoria Josefsson, MDb,c ∙ Hanna Sartor, PhDb,d ∙ David Schmidt, MDb,d ∙ Annie Hjelmgren, MDd ∙ et al. https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(25)02464-X/abstract?dgcid=raven_jbs_etoc_feature_lancet Heart failure with reduced ejection fraction Antonio Cannata, MDa,b ∙ Maria Generosa Crespo-Leiro, MDc ∙ Daniel I Bromage, PhDa,b ∙ Frank Ruschitzka, MDd ∙ Theresa A McDonagh, MD https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(25)01851-3/abstract?dgcid=raven_jbs_etoc_feature_lancet Resident doctors: an investment in the future of the NHS The Lancet https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(26)00193-5/fulltext?dgcid=raven_jbs_etoc_email Jan 31, 2026 Volume 407Number 10527p469-546, e1-e3 https://www.thelancet.com/journals/lancet/issue/vol407no10527/PIIS0140-6736(26)X2002-5

Cutaneous T-Cell Lymphoma: Natural History and the Need for Better Diagnostic and Treatment Efficacy by Madaline Spencer| Published on: Aug 1, 2025 ++

Cutaneous T-Cell Lymphoma: Natural History and the Need for Better Diagnostic and Treatment Efficacy by Madaline Spencer| Published on: Aug 1, 2025 https://checkrare.com/cutaneous-t-cell-lymphoma-natural-history-and-the-need-for-better-diagnostic-and-treatment-efficacy/ New Staging Tool Available for Healthcare Providers Treating Patients With CTCL by CheckRare Staff| Published on: Aug 15, 2025 https://checkrare.com/new-staging-tool-available-for-healthcare-providers-treating-patients-with-ctcl/

2026 Orphan Drugs: PDUFA Dates and FDA Approvals ++++

FDA NEWS 2026 Orphan Drugs: PDUFA Dates and FDA Approvals https://checkrare.com/2026-orphan-drugs-pdufa-dates-and-fda-approvals/ Rare diseases and orphan drugs are at the forefront of novel development and groundbreaking research. Almost half of all novel medications approved by the U.S. Food and Drug Administration (FDA) are orphan drugs. Below is the list of important regulatory dates for all orphan drugs for 2026. FDA Approves Oral Prophylactic Therapy (berotralstat) for Pediatric Patients With Hereditary Angioedema https://checkrare.com/fda-approves-oral-prophylactic-therapy-berotralstat-for-pediatric-patients-with-hereditary-angioedema/ The U.S. FDA has approved Orladeyo (berotralstat) for prophylactic therapy in pediatric patients with hereditary angioedema (HAE) ages 2 to 12 years of age. FDA Approves Gene Therapy (Waskyra) for Patients with Wiskott-Aldrich Syndrome https://checkrare.com/fda-approves-gene-therapy-waskyra-for-patients-with-wiskott-aldrich-syndrome/ The U.S. FDA has approved Waskyra (etuvetidigene autotemcel) for the treatment of patients ages 6 months and older with Wiskott-Aldrich syndrome (WAS). FDA Approves Selumetinib to Treat Patients With Neurofibromatosis Type 1 https://checkrare.com/fda-approves-selumetinib-to-treat-patients-with-neurofibromatosis-type-1/ The U.S. FDA has approved Koselugo (selumetinib) for the treatment of adult patients with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN).

Recognizing and Diagnosing WHIM Syndrome by CheckRare Staff| Updated on: Jan 12, 2026 +++++

RARE DISEASE LEARNING CENTERS Generalized Pustular Psoriasis https://checkrare.com/generalized-pustular-psoriasis/ Generalized pustular psoriasis (GPP) is a rare, severe form of psoriasis characterized by sterile pustules across large areas of skin.  Erdheim-Chester Disease https://checkrare.com/erdheim-chester-disease-2/ Erdheim-Chester disease (ECD) is a rare condition affecting many parts of the body. Although diagnosed in some children, it most commonly affects adults. Cutaneous T-Cell Lymphoma (CTCL) Learning Center https://checkrare.com/cutaneous-t-cell-lymphoma-2/ Cutaneous T-cell lymphoma (CTCL) belongs to the non-Hodgkin lymphoma class of hematologic T-cell lymphoproliferative disorders. What Is WHIM Syndrome? https://checkrare.com/whim-syndrome/ WHIM syndrome is a rare, congenital primary immunodeficiency disorder associated with neutropenia that typically presents in childhood or adolescence. Recognizing and Diagnosing WHIM Syndrome https://checkrare.com/recognizing-and-diagnosing-whim-syndrome/ The features listed in the acronym (W)arts, (H)ypogammaglobulinemia, (I)nfections, and (M)yelokathexis represent the key manifestations of the disorder. However, not all patients will present with the four key features of WHIM at any given time.

Long-Term Data With Dawnzera for the Treatment of Hereditary Angioedema by Madaline Spencer| ++++

CLINICAL PERSPECTIVES Long-Term Data With Dawnzera for the Treatment of Hereditary Angioedema https://checkrare.com/long-term-data-with-dawnzera-for-the-treatment-of-hereditary-angioedema/ Michael Manning, MD, Allergist and Immunologist, discusses long-term data with Dawnzera (donidalorsen) for the treatment of patients with hereditary angioedema (HAE). The Development of Nipocalimab for the Treatment of Sjögren’s Disease https://checkrare.com/the-development-of-nipocalimab-for-the-treatment-of-sjogrens-disease/ Ghaith Noaiseh, MD, Associate Professor of Allergy, Clinical Immunology, and Rheumatology at The University of Kansas Medical Center, discusses the development of nipocalimab for the treatment of Sjögren’s disease (SjD). Post Hoc Analysis of Long-Term Efficacy of Migalastat in Females With Fabry Disease https://checkrare.com/post-hoc-analysis-of-long-term-efficacy-of-migalastat-in-females-with-fabry-disease/ A recent post hoc analysis of long-term efficacy of migalastat in females with Fabry disease was published in the Journal of Medical Genetics. Current Trends in the Healthcare Job Market https://checkrare.com/current-trends-in-the-healthcare-job-market/ Vicki Salemi, Career Expert, discusses current trends in the healthcare job market and expectations for 2026.

Researchers identify new blood markers that may detect early pancreatic cancer NIH-funded, four-marker panel could one day help catch one of deadliest cancers at more treatable stages.

https://www.nih.gov/news-events/news-releases/researchers-identify-new-blood-markers-may-detect-early-pancreatic-cancer RESEARCHERS IDENTIFY NEW BLOOD MARKERS THAT MAY DETECT EARLY PANCREATIC CANCER NIH-funded, four-marker panel could one day help catch one of deadliest cancers at more treatable stages National Institutes of Health (NIH)-supported investigators have developed a blood test to find pancreatic ductal adenocarcinoma, one of the deadliest forms of cancer. The new test could improve survival rates from pancreatic cancer, which tends to be diagnosed at late stages when therapy is less likely to be effective. The findings were published in Clinical Cancer Research. Overall, only about 1 in 10 pancreatic cancer patients survive more than five years from diagnosis. However, experts expect that when the cancer is found and treated at an earlier stage, survival would improve. While finding the cancer early is key, there are no current screening methods to do so. In the study, scientists at the University of Pennsylvania Perelman School of Medicine, Philadelphia, and Mayo Clinic, Rochester, Minnesota, used a phased approach to testing biomarkers in the blood collected from patients with pancreatic cancer and similar patients without the malignancy. They included two blood biomarkers previously explored for use in this way, carbohydrate antigen 19-9 (CA19-9), which is used to monitor treatment response in patients with pancreatic cancer, and thrombospondin 2 (THBS2), another previously used marker. Neither worked well as a screening tool. CA19-9 can be elevated in people with benign conditions such as pancreatitis and bile duct obstruction while other patients don't produce it at all due to genetic factors. In analyzing banked blood samples, the team found two novel biomarker proteins that were elevated in the blood of early-stage pancreatic cancer patients compared with healthy volunteers, aminopeptidase N (ANPEP) and polymeric immunoglobin receptor (PIGR). When they combined ANPEP and PIGR with CA19-9 and THBS2 the four-marker panel successfully distinguished pancreatic cancer cases from non-cases 91.9% of the time for all stages combined at a false positive rate of 5% in non-cases. Similarly, for early-stage (stage I/II) cancer, the four-marker test identified 87.5% of cases. "By adding ANPEP and PIGR to the existing markers, we've significantly improved our ability to detect this cancer when it's most treatable,” said the study’s lead investigator, Kenneth Zaret, Ph.D., University of Pennsylvania's Perelman School of Medicine. Importantly, the four-marker test successfully distinguished cancer patients from both healthy individuals and those with non-cancerous pancreatic conditions, such as pancreatitis. “Our retrospective study findings warrant further testing in larger populations, particularly in people before they show symptoms,” Zaret said. “Such ‘prediagnostic’ studies would help determine if the test could be used as a screening tool for people at high risk of developing the disease based on family history, genetic screening results or personal history of pancreatic cysts or pancreatitis.” REFERENCE: Krusen BM, Gimotty PA, Donahue G, Till JE, Yin M, Carlson EE, Bamlet WR, Carpenter EL, Majumder S, Oberg AL, Zaret KS. “Improving a plasma biomarker panel for early detection of pancreatic ductal adenocarcinoma with aminopeptidase N (ANPEP) and polymeric immunoglobin receptor (PIGR)” Published January 29, 2026, in Clinical Cancer Research. https://aacrjournals.org/clincancerres/article/doi/10.1158/1078-0432.CCR-25-3297/771953/Improving-a-Plasma-Biomarker-Panel-for-Early

Role of Radiotherapy in Rectal Cancer Dominik Modest, MD · Charité – Universitätsmedizin Berlin ++++

https://decisionpoint.medscape.com/oncology/viewarticle/944865?src=0_nl_sm_0&uac=148436CN Key Takeaways: Patients with rectal cancer who are considered for interdisciplinary treatment are typically those with low or middle third tumors. Such tumors are usually up to 12 cm from the anocutaneous line. These rectal cancers have a T3 or T4 magnetic resonance imaging (MRI)-staged tumor and/or nodal positivity. Patients with rectal cancer have historically been treated with radiochemotherapy, followed by surgery and possible adjuvant chemotherapy for an extended period of time. Current treatment strategies, such as those described in the RAPIDO and PRODIGE 23 clinical trials, will most likely include radiotherapy for those individuals at high risk, supplemented with the use of additional systemic therapy. Currently, it is being suggested that patients with low-risk T3 rectal tumors with a small margin and without any metastases in the lymph nodes be treated with surgery and/or chemotherapy in the future, without the need for radiotherapy—that is, the de-escalation of radiotherapy in low-risk advanced rectal cancers. Neoadjuvant Chemotherapy in Colon Cancer: Who Is the Ideal Candidate? Chiara Cremolini, MD, PhD · University of Pisa https://decisionpoint.medscape.com/oncology/viewarticle/944624?src=0_nl_sm_0&uac=148436CN Key Takeaways: The concept of using neoadjuvant chemotherapy is beginning to be considered in colon cancer Neoadjuvant chemotherapy has the potential to shrink micrometastases sufficiently to provide early control of micrometastatic disease through radical surgical intervention The FOxTROT trial revealed some promising data for the use of neoadjuvant chemotherapy in colon cancer, although questions remain about adequate staging of the disease to prevent overtreatment RET Fusions in CRC John L. Marshall, MD · Georgetown University https://decisionpoint.medscape.com/oncology/viewarticle/984929?src=0_nl_sm_0&uac=148436CN Key Takeaways: In mCRC, MSI, RAS, BRAF, and HER2 mutations are generally tested for prior to treatment, and to guide additional therapies; however, RET fusions may also be useful in determining treatment regimens. RET fusions, which involve a chromosome portion fused to another chromosome, can lead to an overproduction signal on a tyrosine kinase gene. RET fusions are thought to be present in only 2% of mCRC patients, however, a recent study suggested selpercatinib led to a 45% response rate in any cancer. Adjuvant Therapy for Small Bowel Adenocarcinoma Katrina S. Pedersen, MS, MD · Washington University https://decisionpoint.medscape.com/oncology/viewarticle/921576?src=0_nl_sm_0&uac=148436CN

What do you do when you’re getting the prescription refill runaround? Trying to refill a medication left me feeling exasperated Written by Tré LaRosa | January 29, 2026 ++++++

What do you do when you’re getting the prescription refill runaround? Trying to refill a medication left me feeling exasperated https://cysticfibrosisnewstoday.com/columns/what-do-when-getting-prescription-refill-runaround/?mc_euid=ffb9b091a2&utm_source=CF&utm_campaign=72530eb696-Email_ENL_UNKNOWN_CF&utm_medium=email&utm_term=0_b075749015-72530eb696-72020733 Written by Tré LaRosa | January 29, 2026 CF treatment Trikafta boosts lung function for patients over 40 Study assesses effects in patients older than those in clinical trials https://cysticfibrosisnewstoday.com/news/cf-treatment-trikafta-boosts-lung-function-patients-40/?mc_euid=ffb9b091a2&utm_source=CF&utm_campaign=72530eb696-Email_ENL_UNKNOWN_CF&utm_medium=email&utm_term=0_b075749015-72530eb696-72020733 Written by Andrea Lobo, PhD | January 27, 2026 Kaftrio may reduce need for salt supplementation in CF, study suggests Treatment restored electrolyte balance, reduced blood carbon dioxide levels https://cysticfibrosisnewstoday.com/news/kaftrio-reduce-need-salt-supplementation-cf-study/?utm_source=CF&utm_campaign=72530eb696-Email_ENL_UNKNOWN_CF&utm_medium=email&utm_term=0_b075749015-72530eb696-72020733 Written by Steve Bryson, PhD | January 22, 2026 Honoring my daughter, the other CF warrior, on her 30th birthday The children of patients deserve recognition, support, and acknowledgment https://cysticfibrosisnewstoday.com/columns/honoring-my-daughter-other-cf-warrior-30th-birthday/?utm_source=CF&utm_campaign=72530eb696-Email_ENL_UNKNOWN_CF&utm_medium=email&utm_term=0_b075749015-72530eb696-72020733 Written by Jennifer Cogliano | January 26, 2026 Turning 40: My once-unattainable goal with CF is now within reach I've been through a war, so getting older is a privilege https://cysticfibrosisnewstoday.com/columns/turning-40-once-unattainable-goal-cf-reach/?utm_source=CF&utm_campaign=72530eb696-Email_ENL_UNKNOWN_CF&utm_medium=email&utm_term=0_b075749015-72530eb696-72020733 Written by Lara Govendo | January 23, 2026 Tiny vesicles boost delivery of fat-soluble vitamins to CF patients: Trial Nanomicelles improve absorption into bloodstream, clinical outcomes https://cysticfibrosisnewstoday.com/news/tiny-vesicles-boost-delivery-fat-soluble-vitamins-cf-patients/?utm_source=CF&utm_campaign=72530eb696-Email_ENL_UNKNOWN_CF&utm_medium=email&utm_term=0_b075749015-72530eb696-72020733 Written by Steve Bryson, PhD | January 26, 2026

What Role Does DNA Analysis Play in Modern Disease Diagnostics? ++++

What role does DNA analysis play in modern disease diagnostics? https://www.azolifesciences.com/whitepaper/20260112/What-role-does-DNA-analysis-play-in-modern-disease-diagnostics.aspx?utm_source=news_medical_newsletter&utm_medium=email&utm_campaign=rare_diseases_newsletter_27_january_2026 What role does DNA analysis play in modern disease diagnostics?DNA analysis is a crucial tool for identifying harmful pathogens, such as bacteria, viruses, or fungi, that cause infections. Unraveling liver injury mechanisms in familial hypobetalipoproteinemia https://www.news-medical.net/news/20260124/Unraveling-liver-injury-mechanisms-in-familial-hypobetalipoproteinemia.aspx?utm_source=news_medical_newsletter&utm_medium=email&utm_campaign=rare_diseases_newsletter_27_january_2026 Unraveling liver injury mechanisms in familial hypobetalipoproteinemiaFamilial Hypobetalipoproteinemia (FHBL), caused by variants in the apolipoprotein B (APOB) gene, is a rare autosomal co-dominant monogenic disorder characterized by lifelong low plasma levels of total cholesterol, low-density lipoprotein cholesterol (LDL-C), and APOB. DLL3 on circulating tumor cells predicts tarlatamab success in lung cancer https://www.news-medical.net/news/20260114/DLL3-on-circulating-tumor-cells-predicts-tarlatamab-success-in-lung-cancer.aspx?utm_source=news_medical_newsletter&utm_medium=email&utm_campaign=rare_diseases_newsletter_27_january_2026 A team led by investigators at the Mass General Brigham Cancer Institute has discovered that a particular marker on tumor cells circulating in the blood indicates whether a patient with lung cancer will experience a lasting response to a newly approved immunotherapy called tarlatamab. UT Southwestern performs first regional whole-liver chemotherapy for rare eye cancer https://www.news-medical.net/news/20260121/UT-Southwestern-performs-first-regional-whole-liver-chemotherapy-for-rare-eye-cancer.aspx?utm_source=news_medical_newsletter&utm_medium=email&utm_campaign=rare_diseases_newsletter_27_january_2026 A team at UT Southwestern Medical Center this week became the first in Texas and neighboring states to successfully perform a novel procedure to deliver whole-liver chemotherapy to treat metastatic uveal melanoma, a rare and deadly eye cancer.

Cancer vaccines enter a new era of personalized immunotherapy ++

Cancer vaccines enter a new era of personalized immunotherapy https://www.news-medical.net/news/20260122/Cancer-vaccines-enter-a-new-era-of-personalized-immunotherapy.aspx?utm_source=news_medical_newsletter&utm_medium=email&utm_campaign=rare_diseases_newsletter_27_january_2026 A new comprehensive review from researchers at the Icahn School of Medicine at Mount Sinai details how decades of cancer vaccine research are converging into a new era of more precise, personalized, and effective immunotherapies, particularly when combined with other cancer treatments. Whole-genome sequencing improves PARP inhibitor treatment prediction https://www.news-medical.net/news/20260112/Whole-genome-sequencing-improves-PARP-inhibitor-treatment-prediction.aspx?utm_source=news_medical_newsletter&utm_medium=email&utm_campaign=rare_diseases_newsletter_27_january_2026 A whole-genome sequencing approach shows early promise over current commercial methods for identifying more patients likely to benefit from PARP inhibitor cancer treatments, according to a study led by Weill Cornell Medicine and NewYork-Presbyterian investigators.

New evidence shows Parkinson’s spreads via immune cells from the gut

https://www.news-medical.net/news/20260129/New-evidence-shows-Parkinsone28099s-spreads-via-immune-cells-from-the-gut.aspx New research reveals how Parkinson's spreads from the gut to the brain, with the help of immune cells – offering a new potential therapeutic strategy – in a study in mice led by scientists at the UK Dementia Research Institute at UCL (University College London).

Rice’s SynthX Center leads interdisciplinary effort to tackle rare lymphatic disorders +++

Rice’s SynthX Center leads interdisciplinary effort to tackle rare lymphatic disorders https://www.news-medical.net/news/20260120/Ricee28099s-SynthX-Center-leads-interdisciplinary-effort-to-tackle-rare-lymphatic-disorders.aspx?utm_source=news_medical_newsletter&utm_medium=email&utm_campaign=rare_diseases_newsletter_27_january_2026 Rice University's SynthX Center, directed by Han Xiao, has received an up to five-year, $18 million award from the Advanced Research Projects Agency for Health (ARPA-H) Lymphatic Imaging, Genomics and pHenotyping Technologies (LIGHT) program to develop innovative solutions for lymphatic diseases. New pangenomic approach enables unprecedented scaling for genetic data https://www.azolifesciences.com/news/20260114/New-Pangenomic-Approach-Enables-Unprecedented-Scaling-for-Genetic-Data.aspx?utm_source=news_medical_newsletter&utm_medium=email&utm_campaign=rare_diseases_newsletter_27_january_2026 Researchers at the University of California have created a novel data structure and compression method that allows pangenomics to process genetic information at previously unattainable scales. FIU's personalized cancer treatment shows promise https://www.news-medical.net/news/20260122/FIUs-personalized-cancer-treatment-shows-promise.aspx?utm_source=news_medical_newsletter&utm_medium=email&utm_campaign=rare_diseases_newsletter_27_january_2026 Researchers at FIU are advancing a personalized approach to cancer treatment that is showing increasingly promising results for patients with hard-to-treat disease, supported by new philanthropic investment that is helping expand the science behind the work.

Edible Insects as Food: Nutritional Benefits, Safety, and Environmental Impact

https://www.news-medical.net/health/Edible-Insects-as-Food-Nutritional-Benefits-Safety-and-Environmental-Impact.aspx Edible insects provide nutrient-dense proteins, beneficial lipids, essential micronutrients, and dietary fiber, with nutritional profiles shaped by species, life stage, processing, and rearing substrates. Current evidence, largely based on compositional analyses, supports their potential role in sustainable food systems while highlighting the need for standardized production, safety oversight, and long-term human studies.

Lifetime heavy drinking sharply raises colorectal cancer risk, major US study finds

https://www.news-medical.net/news/20260128/Lifetime-heavy-drinking-sharply-raises-colorectal-cancer-risk-major-US-study-finds.aspx A large US trial shows that the cumulative burden of alcohol over decades matters for colorectal cancer risk, with heavy lifelong drinking increasing danger and stopping alcohol linked to fewer early precancerous polyps.

Join our Webinar: From Concern to Confidence: Strengthening 988 & 911 Coordination While Managing Risk and Liability February 10, 2–3:30 p.m. ET

https://us06web.zoom.us/webinar/register/WN_dOXie38gQzCjND6jx8mCWQ?utm_source=SAMHSA&utm_campaign=9d5f9516d3-EMAIL_CAMPAIGN_2026_01_26_03_57&utm_medium=email&utm_term=0_-9d5f9516d3-167840245#/registration SAMHSA invites states, counties, Tribal nations, territories, 911 centers, and behavioral health crisis service providers to join a practical, solutions-focused webinar introducing a new framework designed to help communities move forward with confidence.    Key learning objectives: Understand the difference between perceived and actual liability risks in 988 vs.911 coordination Learn how to use Memorandums of Understanding (MOUs), protocols, and training to support safer collaboration Explore risk assessment and triage frameworks that improve outcomes Review real-world state and Tribal examples addressing liability and responder protections Identify clear next steps to strengthen coordination in your own communities

HHS National Conference on Women's Health Date: March 11-13, 2026

https://www.hhs.gov/national-conference-womens-health/index.html?utm_medium=email&utm_source=govdelivery Abstract Submissions for the National Conference on Women’s Health Have Been Extended to February 6! The U.S. Department of Health and Human Services, Office on Women’s Health (OWH) invites you to share your expertise and insights at the Inaugural National Conference on Women’s Health (NCWH 2026), taking place March 11–13, 2026, in Washington, D.C.! Due to overwhelming excitement surrounding poster abstract submissions, the deadline to submit has been extended! Attendees are encouraged to submit an abstract to be considered for a poster by February 6, 11:59 p.m. ET. Please review the abstract submission instructions prior to submitting your abstract for consideration. Whether your work focuses on research, clinical practice, or public health initiatives, we want to hear from you. Accepted abstracts will be featured as poster presentations during dedicated sessions at the conference. https://vfairs-core-backend-prod.s3.amazonaws.com/20362/document/File-1768591242.pdf?utm_medium=email&utm_source=govdelivery Topics of interest include: Preventing and Reversing Chronic Diseases in Women and Girls Addressing Sex as a Biological Variable in Physical and Mental Health Understanding the Female Cycle as a Vital Sign Optimizing Health during Menopause Ready to submit? 👉 Visit the NCWH 2026 abstract submission page for details and formatting guidelines: https://ncwh2026.com/en/abstract-information. https://ncwh2026.com/en/abstract-information?utm_medium=email&utm_source=govdelivery Why participate? Showcase your work to a national audience of health care professionals Contribute to the evidence base advancing women’s health Network with peers and leaders in the field Final reminders: Abstract deadline: February 6, 2026 Conference dates: March 11–13, 2026 Location: Omni Shoreham Hotel, Washington, D.C. Last day to book with the discounted rate: February 17, 2026 We look forward to receiving your abstract and seeing you in March!

Health Benefits and Nutritional Value of Mamey Sapote +++

Health Benefits and Nutritional Value of Mamey Sapote https://www.news-medical.net/health/Health-Benefits-and-Nutritional-Value-of-Mamey-Sapote.aspx From traditional Mesoamerican staple to emerging functional food, mamey sapote offers compelling nutritional promise while science works to define the boundaries of its proven health benefits. Kokum Fruit: Nutrition, Health Benefits, and Scientific Evidence https://www.news-medical.net/health/Kokum-Fruit-Nutrition-Health-Benefits-and-Scientific-Evidence.aspx Kokum (Garcinia indica) is a traditionally used Indian fruit increasingly explored in modern nutrition for its bioactive compounds, antioxidant capacity, and role in functional foods. Current evidence is largely preclinical, with limited human data supporting cautious interpretation of its metabolic and digestive health potential. Camel Milk Nutrition Facts and Potential Health Benefits Explained https://www.news-medical.net/health/Camel-Milk-Nutrition-Facts-and-Potential-Health-Benefits-Explained.aspx Camel milk is a nutrient-dense dairy alternative with distinct protein composition, bioactive compounds, and potential metabolic and immune-supporting properties. Current evidence suggests benefits for digestibility and glucose regulation, while emphasizing the need for larger, standardized clinical trials to confirm therapeutic effects.

Color vision deficiency is linked to worse survival in bladder cancer

https://www.news-medical.net/news/20260128/Color-vision-deficiency-is-linked-to-worse-survival-in-bladder-cancer.aspx A large electronic health record study suggests that difficulty recognizing painless blood in urine may delay diagnosis and worsen long-term survival for bladder cancer patients with color vision deficiency, highlighting overlooked risks in symptom recognition and screening.

FDA Releases New Interactive Tool for Total Diet Study (TDS) Results and New Data

https://www.fda.gov/food/hfp-constituent-updates/fda-releases-new-interactive-tool-total-diet-study-tds-results-and-new-data?utm_medium=email&utm_source=govdelivery January 27, 2026 The FDA today announced the release of the Total Diet Study Interface (TDSi), a new consumer-friendly interactive web-based tool that provides streamlined access to findings from FDA's Total Diet Study. Concurrent with this launch, the FDA is releasing the most recent cycle of TDS data, including results from fiscal years 2021 and 2022. TDSi includes TDS datasets from fiscal year 2018–2022. https://www.fda.gov/food/reference-databases-and-monitoring-programs-food/fda-total-diet-study-tds?utm_medium=email&utm_source=govdelivery The TDSi release underscores the FDA's commitment to transparency and continuous improvement in its food chemical safety programs and aligns with Make America Healthy Again goals. By transitioning from traditional publication methods to this dynamic, user-interactive platform, the FDA is providing stakeholders with significantly enhanced access to critical food safety and nutrition data, positioning itself as the global leader in providing comprehensive, publicly accessible data on foods in a typical U.S. consumer’s diet. https://www.hhs.gov/radical-transparency/index.html?utm_medium=email&utm_source=govdelivery FDA's TDS is a continuous monitoring program that systematically collects, prepares and analyzes chemical contaminants and nutrients in foods representing the average U.S. consumer's diet. This new enhancement to the TDS surveillance program provides critical data to assess cumulative exposure to various contaminants and supports the FDA's food safety and nutrition oversight responsibilities. The TDSi represents a significant advancement in data accessibility and transparency, offering: Comprehensive data visualization – Interactive graphics and charts that enable users to easily explore TDS findings efficiently and intuitively. Enhanced transparency – User-driven access to detailed analytical results with full dataset download capabilities for researchers and stakeholders. Historical scope – Complete datasets for nutrients and contaminants (including elements, radionuclides, and pesticides) in foods collected since 2018. Ongoing updates – Regular incorporation of new TDS data as it becomes available. For comprehensive information on TDS design and implementation, foods and dietary exposure estimation, and analytical methods, visit the FDA TDS webpage. https://www.fda.gov/food/food-ingredients-packaging/food-chemical-safety?utm_medium=email&utm_source=govdelivery

Strengthening cardiovascular disease prevention in Europe The Lancet Public Health ++... ++

Strengthening cardiovascular disease prevention in Europe The Lancet Public Health https://www.thelancet.com/journals/lanpub/article/PIIS2468-2667(26)00007-1/fulltext?dgcid=raven_jbs_etoc_email Feb 2026 Volume 11Number 2e69-e136 https://www.thelancet.com/journals/lanpub/issue/vol11no2/PIIS2468-2667(26)X2001-1 Uptake and performance of self-collection offered through primary care to all eligible participants in a national cervical screening programme in Australia: a retrospective cohort study Farhana Sultana, PhDa ∙ Megan Smith, PhDb ∙ Prof Marion Saville, MBChBc,d ∙ Prof Deborah Bateson, MB BSf ∙ Prof David Roder, DDScg ∙ Vivienne Milch, MHPolh,i ∙ et al. https://www.thelancet.com/journals/lanpub/article/PIIS2468-2667(25)00304-4/fulltext?dgcid=raven_jbs_etoc_feature_lanpub The potential effect of a geographically focused intervention against tuberculosis in the USA: a simulation modelling study Mathilda Regan, PhDa mathildaregan@hsph.harvard.edu ∙ Hening Cui, MSa ∙ Nicole A Swartwood, MSPHa ∙ Yunfei Li, ScDa ∙ Suzanne M Marks, MPHb ∙ Terrika Barham, PhDc ∙ et al. https://www.thelancet.com/journals/lanpub/article/PIIS2468-2667(25)00306-8/fulltext?dgcid=raven_jbs_etoc_feature_lanpub

Unite With the Rare Disease Community to Create Powerful Partnerships, Advance Orphan Therapeutics and Deliver Life-Changing Breakthroughs March 17-19, 2026 Pre-Conference Workshops on March 16, 2026

https://informaconnect.com/rare-disease-summit/ Don’t miss the Rare Disease Summit, March 17-19 in Philadelphia, PA, United States. You'll connect with key stakeholders to create powerful partnerships, advance orphan therapeutics and inspire advocacy. 60+ expert speakers, including the FDA and Pharma/Bio execs, will discuss critical rare disease breakthroughs, and you’ll return to your office having gained solution-focused strategies surrounding patient access, reimbursement commercialization, launch partnering and more. You'll hear from manufacturers, patients, advocates and regulators, as they cover content on the hottest topics. Engage in sessions on the challenges and opportunities surrounding the AI Ecosystem, Pre-Launch Strategy, Healthcare Policy, Patient Access and Advocacy, Affordability, Patient-Physician Communication, Payer Partnerships, Post-Launch Execution, Navigating the FDA Landscape, U.S. and Global Launch, Cell and Gene Trials, Crafting BD Strategy and and much more! Don’t miss the chance to come together with colleagues from around the globe to unite in areas of unmet medical need and create life-transforming therapies and breakthroughs. Learn more at www.informaconnect.com/rare.

FDA approves daratumumab and hyaluronidase-fihj with bortezomib, lenalidomide, and dexamethasone for newly diagnosed multiple myeloma

https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-daratumumab-and-hyaluronidase-fihj-bortezomib-lenalidomide-and-dexamethasone-newly On January 27, 2026, the Food and Drug Administration approved daratumumab and hyaluronidase-fihj (Darzalex Faspro, Janssen Biotech, Inc.) in combination with bortezomib, lenalidomide, and dexamethasone (VRd) for adults with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant (ASCT).

Cellular mitochondria transfer prevents pain

https://www.nih.gov/news-events/nih-research-matters/cellular-mitochondria-transfer-prevents-pain At a Glance Studies in mice and human cells revealed that power-hungry sensory neurons get mitochondria for energy production from nearby supporting cells. The results point to potential new treatments for nerve pain caused by drugs or health conditions that harm mitochondria.

ADHD medications stimulate alertness, motivation

https://www.nih.gov/news-events/nih-research-matters/adhd-medications-stimulate-alertness-motivation At a Glance Researchers found that prescription stimulants for ADHD act on brain networks that control wakefulness and reward, but not attention as previously thought. The study suggests that stimulants and additional sleep affect the brain in similar ways, and that getting enough sleep could help in managing ADHD.

Testing risk-based breast cancer screening

https://www.nih.gov/news-events/nih-research-matters/testing-risk-based-breast-cancer-screening At a Glance In a large clinical trial, risk-based breast cancer screening was as safe and effective as annual mammograms. This approach could help reduce anxiety, costs, and unnecessary follow-up testing.

Join us for NCSACW’s upcoming webinar: Strengthening Father Engagement to Improve Child and Family Outcomes Wednesday, February 4, 2–3 p.m. ET (11 a.m.–12 p.m. PT)

https://cffutures.zoom.us/webinar/register/WN_SDGlJzHwTQWbM0jIMSMsOA?utm_source=SAMHSA&utm_campaign=b4edd3d99e-EMAIL_CAMPAIGN_2026_01_26_05_46_COPY_01&utm_medium=email&utm_term=0_-a947656de7-167840245#/registration Strong families remain a cornerstone of a healthy society and, fathers play a crucial role—especially in the areas of child safety, stability, and well-being. Children with involved fathers experience stronger developmental foundations and lower risks for poor outcomes, such as academic challenges, justice system involvement, substance use, and infant mortality. However, fathers with substance use disorders (SUD) who are also involved with child welfare often experience organizational practices and procedures designed for mothers. This webinar: Explores the importance of engaging fathers Offers attendees practical strategies to overcome common barriers to engagement in child welfare, SUD treatment, and court services Demonstrates how these efforts strengthen family support systems and lead to improved outcomes—such as increased reunification, reduced risk of future maltreatment allegations, and shorter stays in foster care The session will provide strategies to fully integrate fathers into services while increasing safety factors for children and families affected by substance use and child welfare involvement. Attendees will: Recognize the importance of: 1) including fathers in child welfare and treatment case planning, and 2) promoting father-child relationships Learn effective strategies to engage fathers in treatment and services Identify ways to promote father engagement within child welfare, SUD treatment, and court systems Meet our presenter: Robert Rosenhaus, MSW, Center for Children and Family Futures Senior Program Associate, Regional Partnership Grants Program

The patient impact of 11 years of ocrelizumab treatment in multiple sclerosis: long-term data from the Phase III OPERA and ORATORIO studies

The patient impact of 11 years of ocrelizumab treatment in multiple sclerosis: long-term data from the Phase III OPERA and ORATORIO studies Early and sustained treatment of patients with MS with a high efficacy therapy delays disability progression and the time to reaching key long-term disability milestones. OCR has a robust long-term safety and efficacy experience across the MS spectrum. Results for OCR over eleven years are reported for 48-week CDP and other measures of progression (including the rate of repeated 48-week CDP, and the time to requiring walking aid or wheelchair) during OPERA I/II (NCT01247324/NCT01412333), ORATORIO (NCT01194570) and their open-label extensions. https://medically.roche.com/global/en/medical-material.7539ff6d-3fbb-422c-bd07-6b2f2616a529.qr.html?cid=slpsectrims20242409xxnvspht

Does vitamin D supplementation meaningfully reduce mortality or length of stay in sepsis (Medscape AI)

https://www.medscape.com/ai-search?query=Does%20vitamin%20D%20supplementation%20meaningfully%20reduce%20mortality%20or%20length%20of%20stay%20in%20sepsis&%20&ecd=wnl_tp10_daily_260123_MSCPEDIT_etid8049873&uac=148436CN&impID=8049873 In sepsis, direct interventional data on vitamin D supplementation and its impact on mortality or hospital length of stay are lacking. However, observational and meta-analytic data link vitamin D deficiency with worse outcomes in critically ill patients, suggesting a potential but unproven role for supplementation.

How to manage amiodarone-induced thyrotoxicosis? (Medscape AI)

https://www.medscape.com/ai-search?query=How%20to%20manage%20amiodarone-induced%20thyrotoxicosis%3f&ecd=wnl_tp10_daily_260125_MSCPEDIT_etid8053426&uac=148436CN&impID=8053426 Amiodarone-induced thyrotoxicosis (AIT) requires prompt differentiation of its two main subtypes and tailored therapy to mitigate the risk of cardiovascular complications, especially in patients with underlying cardiac disease.

Repeated head trauma causes neuron loss and inflammation in young athletes

https://pubmed.ncbi.nlm.nih.gov/40963024/ NIH STUDY CLEARLY TIES RISK OF DEMENTIA TO SEVERE CTE A study funded by the National Institutes of Health (NIH) provides the clearest evidence to date to link severe chronic traumatic encephalopathy (CTE) to dementia risk. CTE is a degenerative brain disorder in some people who have had repeated head impacts over time. It can only be diagnosed after death by examining brain tissue. While researchers were able to link severe CTE (stages III and IV) to dementia risk, they did not find any measurable link between less severe CTE (stages I and II) and changes in thinking, mood, or daily functioning. The research, led by scientists at Boston University CTE Center and the U.S. Department of Veterans Affairs Boston Healthcare System, analyzed 614 donated brains from people with known exposure to repetitive head impacts. None of the donors had Alzheimer’s disease, Lewy body disease, or frontotemporal lobar degeneration, three of the most common neurodegenerative diseases that cause dementia. CTE is marked by the buildup of abnormal tau protein – which in healthy brains helps keep nerve cells stable and working properly. In CTE, tau forms small, brownish tangles clustered around tiny blood vessels deep in the brain’s grooves and spreads more widely as the disease advances. “By examining hundreds of brains and ruling out other common neurodegenerative diseases, the team could look at CTE alone and linked it to symptoms reported during life,” said Dr. Amy Bany Adams, acting director of the NIH’s National Institute of Neurological Disorders and Stroke (NINDS), which funded the research. The researchers found that individuals with stage IV CTE were 4.5 times more likely to have had dementia during life than donors without CTE. Stage III CTE was also associated with higher dementia risk. In contrast, stages I-II were not found to be associated with dementia, cognitive impairment, or functional decline. Mood and behavioral symptoms were not observed at any stage of CTE. Dr. Richard Hodes, director of the NIH’s National Institute on Aging (NIA), which also funded the research, noted important implications for dementia research. “Understanding which brain changes drive cognitive decline is essential,” he said. “This study shows that only severe CTE has a clear link to dementia, which provides an important distinction for researchers, healthcare providers, and families.” The study also indicates that mood or behavior changes commonly attributed to CTE may instead come about from other brain effects of repetitive head impacts or from unrelated medical or environmental factors. The new results build on NIH-funded work published earlier this year showing that repetitive head impacts in young athletes can trigger early cellular changes – such as immune activation, blood vessel alterations, and loss of certain brain cells – well before tau buildup in the brain appears. However, it is still not clear whether these early brain changes cause any symptoms or lead to CTE later in life. Together, the two studies outline a more complete picture of how repetitive head impacts affect the brain over time. This work was supported by grants from NINDS and NIA. Long-term federal investment enabled analysis of one of the largest CTE cohorts ever assembled, allowing researchers to untangle the specific effects of CTE on brain function and disease for the first time.

Rare Disease Focus: Autoimmune and Auto-Inflammatory Disorders ++ ++ +++++ + +++++

January is Thyroid Eye Disease Awareness Month Thyroid Eye Disease: Overview, Diagnosis, and Current and Emerging Treatment Options https://checkrare.com/thyroid-eye-disease-overview-and-diagnosis/ Thyroid eye disease (TED) is a chronic endocrine, autoimmune disease characterized by immune-mediated orbital inflammation that greatly impacts a person’s vision. How the TED Community Organization Helps Patients With Thyroid Eye Disease Take Their Lives Back https://checkrare.com/how-the-ted-community-organization-helps-patients-with-thyroid-eye-disease-take-their-lives-back/ Christine Gustafson, Executive Director and CEO of the TED Community Organization, discusses her personal journey with thyroid eye disease (TED) and why she started the TED Community Organization. RESOURCES Rare Disease Recognition and Awareness Days https://checkrare.com/rare-disease-awareness-days-and-recognition-page/ Stay informed with our comprehensive calendar of rare disease awareness days, highlighting key dates and resources. Priority Review Voucher Tracking https://checkrare.com/priority-review-voucher/ The Priority Review Voucher (PRV) program incentivizes the development of drugs for rare pediatric and tropical diseases. FDA NEWS 2026 Orphan Drugs: PDUFA Dates and FDA Approvals https://checkrare.com/2026-orphan-drugs-pdufa-dates-and-fda-approvals/ Rare diseases and orphan drugs are at the forefront of novel development and groundbreaking research. Almost half of all novel medications approved by the U.S. Food and Drug Administration (FDA) are orphan drugs. FDA Approves Uplizna for the Treatment of Generalized Myasthenia Gravis https://checkrare.com/fda-approves-uplizna-for-the-treatment-of-generalized-myasthenia-gravis/ The U.S. FDA has approved Uplizna (inebilizumab) for the treatment of generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) and anti-muscle specific tyrosine kinase (MuSK) antibody positive. FDA Approves Oral Prophylactic Therapy (berotralstat) for Pediatric Patients With Hereditary Angioedema https://checkrare.com/fda-approves-oral-prophylactic-therapy-berotralstat-for-pediatric-patients-with-hereditary-angioedema/ The U.S FDA has approved Orladeyo (berotralstat) for prophylactic therapy in pediatric patients with hereditary angioedema (HAE) ages 2 to 12 years of age. FDA Approves Gene Therapy (Waskyra) for Patients with Wiskott-Aldrich Syndrome https://checkrare.com/fda-approves-gene-therapy-waskyra-for-patients-with-wiskott-aldrich-syndrome/ The U.S. FDA has approved Waskyra (etuvetidigene autotemcel) for the treatment of patients ages 6 months and older with Wiskott-Aldrich syndrome (WAS). FDA Approves Voyxact for Primary Immunoglobulin A Nephropathy https://checkrare.com/fda-approves-voyxact-for-primary-immunoglobulin-a-nephropathy/ The U.S. FDA has approved Voyxact (sibeprenlimab) for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk for disease progression. FEATURED ARTICLE A New Test for Patient-Reporting of Mastocytosis Control https://checkrare.com/a-new-test-for-patient-reporting-of-mastocytosis-control/ A team of allergists, immunologists, and dermatologists sought to test a new patient-reported outcomes measure for assessing mastocytosis disease control. Their newly developed tool, called the Mastocytosis Control Test (MCT), was generated through interviews with adult patients with nonadvanced indolent systemic mastocytosis. CLINICAL PERSPECTIVES Long-Term Data With Dawnzera for the Treatment of Hereditary Angioedema https://checkrare.com/long-term-data-with-dawnzera-for-the-treatment-of-hereditary-angioedema/ Michael Manning, MD, Allergist and Immunologist, discusses long-term data with Dawnzera (donidalorsen) for the treatment of patients with hereditary angioedema (HAE). Advancing Thyroid Eye Disease Care: Integrating Tepezza, New Technology, and Optimizing Multidisciplinary Teams https://checkrare.com/advancing-thyroid-eye-disease-care-integrating-tepezza-new-technology-and-optimizing-multidisciplinary-teams/ Maja Kostic, MD, PhD, neuro-ophthalmologist at Bascom Palmer Eye Institute and Assistant Professor of Ophthalmology at the University of North Carolina at Chapel Hill, discusses advancing thyroid eye disease (TED) care. One-Year Phase 3 Data Testing Empaveli (pegcetacoplan) in Patients with C3G and IC-MPGN https://checkrare.com/one-year-phase-3-data-testing-empaveli-pegcetacoplan-in-patients-with-c3g-and-ic-mpgn/ Carla Nester, MD, Professor of Pediatrics-Nephrology at the University of Iowa, discusses one-year phase 3 data of Empaveli (pegcetacoplan) for treatment of C3 glomerulopathy (C3G) and immune complex membranoproliferative glomerulonephritis (IC-MPGN). Interim Results From Study of Mezagitamab for the Treatment of IgA Nephropathy https://checkrare.com/interim-results-from-study-of-mezagitamab-for-the-treatment-of-iga-nephropathy/ Jonathan Barratt, Mayer Professor of Renal Medicine at the University of Leicester, discusses interim results from a study testing mezagitamab for the treatment of immunoglobulin A (IgA) nephropathy. The Development of Nipocalimab for the Treatment of Sjögren’s Disease https://checkrare.com/the-development-of-nipocalimab-for-the-treatment-of-sjogrens-disease/ Ghaith Noaiseh, MD, Associate Professor of Allergy, Clinical Immunology, and Rheumatology at The University of Kansas Medical Center, discusses the development of nipocalimab for the treatment of Sjögren’s disease (SjD).

SUD 101 Webinar Wednesday, January 28, 12 p.m. ET // Pathways to SUD Treatment & Recovery Thursday, January 29, 1 p.m. ET

SUD 101 Webinar Wednesday, January 28, 12 p.m. ET Register for SUD 101 https://capconcorp.zoom.us/webinar/register/WN_Ybj35AN4Rt-lopQE2JricA?utm_source=SAMHSA&utm_campaign=b28ebce7b1-EMAIL_CAMPAIGN_2026_01_26_06_37&utm_medium=email&utm_term=0_-b28ebce7b1-167840245#/registration “Treatment works! Find the path that works for you,” has been the theme for this year’s annual, national SUD Treatment Month. Karran Phillips, M.D., MSc, the acting director of SAMHSA’s Center for Substance Abuse Treatment, will present on SUD treatment options and on the work SAMHSA is doing to facilitate greater access to treatment and to more fully equip medical professionals in position to provide that treatment. Pathways to SUD Treatment & Recovery Thursday, January 29, 1 p.m. ET Register for Pathways to SUD https://capconcorp.zoom.us/webinar/register/WN_QHnpo8kqRgKEkhWaRej2Zg?utm_source=SAMHSA&utm_campaign=b28ebce7b1-EMAIL_CAMPAIGN_2026_01_26_06_37&utm_medium=email&utm_term=0_-b28ebce7b1-167840245#/registration Berlina-Wallace Berube, MACP, MEd, LCPC, NCC, will share information on multiple treatment protocols and recovery supports—all with the goal of helping people find the most appropriate means to attaining and retaining recovery. That will be immediately followed by a fireside chat between Angela Caldwell, CSC-AD, MS, and David Awadalla, MSW, BSHP, SAMHSA experts working in the Center for Substance Abuse Treatment and the Office of Recovery, respectively. The duo will discuss the evolution of the concept of recovery, as well as how centering individuals who have SUD during the treatment process can make a world of difference in their recovery efforts’ success.

Join us for NCSACW’s upcoming webinar: Strengthening Father Engagement to Improve Child and Family Outcomes Wednesday, February 4, 2–3 p.m. ET (11 a.m.–12 p.m. PT)

https://cffutures.zoom.us/webinar/register/WN_SDGlJzHwTQWbM0jIMSMsOA?utm_source=SAMHSA&utm_campaign=a947656de7-EMAIL_CAMPAIGN_2026_01_26_05_46&utm_medium=email&utm_term=0_-a947656de7-167840245#/registration Strong families remain a cornerstone of a healthy society and, fathers play a crucial role—especially in the areas of child safety, stability, and well-being. Children with involved fathers experience stronger developmental foundations and lower risks for poor outcomes, such as academic challenges, justice system involvement, substance use, and infant mortality. However, fathers with substance use disorders (SUD) who are also involved with child welfare often experience organizational practices and procedures designed for mothers. This webinar: Explores the importance of engaging fathers Offers attendees practical strategies to overcome common barriers to engagement in child welfare, SUD treatment, and court services Demonstrates how these efforts strengthen family support systems and lead to improved outcomes—such as increased reunification, reduced risk of future maltreatment allegations, and shorter stays in foster care The session will provide strategies to fully integrate fathers into services while increasing safety factors for children and families affected by substance use and child welfare involvement. Attendees will: Recognize the importance of: 1) including fathers in child welfare and treatment case planning, and 2) promoting father-child relationships Learn effective strategies to engage fathers in treatment and services Identify ways to promote father engagement within child welfare, SUD treatment, and court systems Meet our presenter: Robert Rosenhaus, MSW, Center for Children and Family Futures Senior Program Associate, Regional Partnership Grants Program

What is the recommended interval for cervical cancer screening? (Medscape AI) +... ++++

https://www.medscape.com/ai-search?query=What%20is%20the%20recommended%20interval%20for%20cervical%20cancer%20screening%3F&&ecd=mkm_ret_260127_mscpmrk-OUS_InFocus_etid8050601&uac=148436CN&impID=8050601 HPV Vaccines Have Had a ‘Dramatic’ Impact on Cervical Cancer Ernie Mundell https://www.medscape.com/viewarticle/hpv-vaccines-have-had-dramatic-impact-cervical-cancer-2026a10000jq?ecd=mkm_ret_260127_mscpmrk-OUS_InFocus_etid8050601&uac=148436CN&impID=8050601 January 08, 2026 Women Can Self-test At Home for Cervical Cancer Virus, New Guidelines Say Nancy Lapid https://www.medscape.com/s/viewarticle/women-can-self-test-home-cervical-cancer-virus-new-2025a1000y0h?ecd=mkm_ret_260127_mscpmrk-OUS_InFocus_etid8050601&uac=148436CN&impID=8050601 December 04, 2025 Human Papillomavirus Self-Sampling Linked to Increased Cervical Cancer Screening in Long-Term Non-Attenders Edited by Vineeta Teotia https://www.medscape.com/viewarticle/human-papillomavirus-self-sampling-linked-increased-cervical-2026a10000l2?ecd=mkm_ret_260127_mscpmrk-OUS_InFocus_etid8050601&uac=148436CN&impID=8050601 Medscape UK January 12, 2026 Lower Risk Groups Can Cut Back on Cervical Cancer Screening Heidi Splete https://www.medscape.com/viewarticle/lower-risk-groups-can-cut-back-cervical-cancer-screening-2025a1000ygy?ecd=mkm_ret_260127_mscpmrk-OUS_InFocus_etid8050601&uac=148436CN&impID=8050601 December 09, 2025