CIENCIASMEDICASNEWS: mayo 2025

https://www.thelancet.com/journals/lanwpc/article/PIIS2666-6065(25)00123-3/fulltext?dgcid=raven_jbs_etoc_email TheLancet.com will be undergoing maintenance on Saturday, May 31st from 8am to 12pm US Eastern (4 hours). During this timeframe, we ask that you refrain from logging in or registering to avoid any difficulty with this functionality. We apologise for the inconvenience. May 2025 Volume 58 https://www.thelancet.com/journals/lanwpc/issue/vol58nonull/PIIS2666-6065(25)X0006-7

Measuring ageism +...

https://www.thelancet.com/journals/lanhl/article/PIIS2666-7568(25)00046-7/fulltext?dgcid=raven_jbs_etoc_email TheLancet.com will be undergoing maintenance on Saturday, May 31st from 8am to 12pm US Eastern (4 hours). During this timeframe, we ask that you refrain from logging in or registering to avoid any difficulty with this functionality. We apologise for the inconvenience. May 2025 Volume 6Number 5 https://www.thelancet.com/journals/lanhl/issue/vol6no5/PIIS2666-7568(25)X0006-4

Observed and hidden factors underlying the accumulation of chronic diseases across eight major organ systems: a longitudinal birth cohort study

https://www.thelancet.com/journals/lanhl/article/PIIS2666-7568(25)00029-7/fulltext?dgcid=raven_jbs_etoc_feature_lanhl TheLancet.com will be undergoing maintenance on Saturday, May 31st from 8am to 12pm US Eastern (4 hours). During this timeframe, we ask that you refrain from logging in or registering to avoid any difficulty with this functionality. We apologise for the inconvenience.v

Women and Health: Uncovering Health-Care Disparities

https://www.thelancet.com/video-do/women-and-health?dgcid=raven_jbs_etoc_feature_infocuswomenhealth-video TheLancet.com will be undergoing maintenance on Saturday, May 31st from 8am to 12pm US Eastern (4 hours). During this timeframe, we ask that you refrain from logging in or registering to avoid any difficulty with this functionality. We apologise for the inconvenience. Women and Health: Uncovering Health-Care Disparities In this video, health-care professionals, researchers, charity founders, and Lancet Editors from various fields examine the disparities, frequently exacerbated by intersectionality, that impact women globally.

Next week: Get expert insights into primary care—Thurs. June 5 at 8:00 AM ET

https://www.dhaj7-cepo.com/content/2025-jun-ccss?utm_source=adobe&utm_medium=email&utm_campaign=may302025 Virtual Event NEXT WEEK | Thursday, June 5 @ 8:00 AM ET Join health experts as they highlight innovative strategies and uncover important issues affecting primary care for military and veteran populations. Sessions dive into critical topics, such as the anti-obesity medication landscape, preventing threats from multidrug-resistant organisms, and ethical considerations in multi-cancer early detection testing. Event Sessions Session 01: Slimming Down the Options: A Prescriber’s Guide to Antiobesity Medications 8:15 AM–9:15 AM ET | 1.00 CE/CME credit Brittany L. Howard, PA-C Program Director, Bariatric and Metabolic Surgical Services, Womack Army Medical Center ------------------------------ Session 02: Multidrug-Resistant Organisms: Threats and Prevention Strategies 9:25 AM–10:25 AM ET | 1.00 CE/CME credit Army Maj. Benjamin Custer, M.D., MPH&TM Chair, Infection Prevention and Control, Walter Reed National Military Medical Center; Assistant Professor of Medicine, Uniformed Services University of the Health Sciences ------------------------------ Session 03: Biosimilar and Interchangeable Biological Products: An Overview of Scientific Concepts and Practical Resources 10:35 AM–11:35 AM ET | 1.00 CE/CME credit Sabiha Khan, M.D. Medical Officer, Office of Therapeutic Biologics and Biosimilars, U.S. Food and Drug Administration Sarah Crowley-Ikenberry, M.A. Associate Director, Stakeholder Outreach and Education, Office of Therapeutic Biologic and Biosimilars, U.S. Food and Drug Administration ------------------------------ Session 04: Artificial Intelligence in Primary Care: From Hype to Practical Impact 11:45 AM–12:45 PM ET | 1.00 CE/CME credit Steven Lin, M.D. Clinical Professor, General Primary Care Chief, and Stanford Healthcare AI Applied Research Team Director, Division of Primary Care and Population Health, Stanford University School of Medicine ------------------------------ Session 05: Practical Application of the VA/DOD Clinical Practice Guidelines for Tinnitus in Daily Practice 1:45 PM–2:45 PM ET | 1.00 CE/CME credit LaGuinn P. Sherlock, Au.D., CCC-A Research Audiologist, Defense Centers for Public Health – Aberdeen, and Walter Reed National Military Medical Center Sarah Theodoroff, Ph.D., CCC-A Interim Associate Director, VA National Center for Rehabilitative Auditory Research; Associate Professor, Department of Otolaryngology – Head & Neck Surgery Oregon Health & Science University ------------------------------ Session 06: Ethical Considerations for Multi-Cancer Early Detection Testing 3:00 PM–4:00 PM ET | 1.00 CE/CME credit Navy Capt. Harlan Dorey, M.D., FAAP Officer in Charge, Navy Medicine Readiness Training Unit Bangor; Director of Branch Clinic, Navy Medicine Readiness and Training Command Bremerton; DHA Primary Care Clinical Community Chair

ADHD, Obesity Link May Depend on Where You Live ++++++++++

The Rise of Protein Degraders for Hematologic Malignancies: Exploring the Latest Data From ASH 2024 Authors: Sagar Lonial, MD, FACP; Constatine Tam, MBBS, MD, FRACP, FRCPath; Julio Chavez, MD, MS

https://www.medscape.org/viewarticle/1002295?sso=true&uac=148436CN&src=mkmcmr_driv_stan_mscpedu_250530-OUS-HONC-1002295-cta

Dangerous Trend or Rite of Passage? +++ +++++ ++++

Sharing Expert Minds in Lung Cancer and Lung Neuroendocrine Tumors Delivering Optimal Patient Care Authors: Martin Reck, MD, PhD; Catherine Bouvier; Melanie Lucas; Abdul Masoor, MBChB, PhD, MRCP; Marianne Pavel, MD

https://www.medscape.org/viewarticle/1002284?sso=true&uac=148436CN&src=mkmcmr_driv_stan_mscpedu_250531-OUS-HONC-1002284-cta

viernes, 30 de mayo de 2025

Forging a new path in Hodgkin lymphoma treatment

https://www.statnews.com/sponsor/2025/05/30/forging-a-new-path-in-hodgkin-lymphoma-treatment/

Moderna’s vaccine hit, the MAHA report, and an ASCO preview

https://www.statnews.com/2025/05/29/modernas-vaccine-hit-the-maha-report-and-an-asco-preview/ Summit shows mixed results from key study of lung cancer drug Ivonescimab has been closely tracked for its blockbuster potential https://www.statnews.com/2025/05/30/summit-shows-ivonescimab-lung-cancer-mixed-results/

Next-gen COPD drug from Sanofi, Regeneron shows mixed results in Phase 3 trials The companies are looking for their next Dupixent

https://www.statnews.com/2025/05/30/itepekimab-sanofi-regeneron-copd-dupixent-aerify/

Join SAMHSA’s Webinar on Supporting Young Adults Experiencing Early Serious Mental Illness in the Shift from Child to Adult Mental Health Services Tuesday, June 17, 12–1 p.m. ET

https://westat.zoomgov.com/webinar/register/WN_gr-hJc1hT6SMIl74LZ6QIw?utm_source=SAMHSA&utm_campaign=2b9484b310-EMAIL_CAMPAIGN_2025_05_28_03_47&utm_medium=email&utm_term=0_-2b9484b310-167840245#/registration Join SAMHSA’s Webinar on Supporting Young Adults Experiencing Early Serious Mental Illness in the Shift from Child to Adult Mental Health Services Tuesday, June 17, 12–1 p.m. ET The Substance Abuse and Mental Health Services Administration National Training and Technical Assistance Center for Early Serious Mental Illness (SAMHSA ESMI TTA Center) is hosting a webinar series that addresses a range of topics related to early serious mental illness (ESMI), including clinical high risk for psychosis (CHR-P) and first episode psychosis (FEP). This one-hour webinar will discuss ways that young adults experiencing ESMI may be supported as they shift from child to adult mental health services. Participants will learn about structural and procedural changes that occur as young adults shift out of ESMI services and move from child to adult service systems. The webinar will also identify promising strategies to reduce service gaps, including using early planning, assessment tools, cross-system coordination, and personalized step-down programs. Two grantees will present approaches to improve continuity of care, minimize disengagement, and enhance service availability for young adults experiencing ESMI. Featured Presenters: Kathryn Sabella, Ph.D., Assistant Professor, Department of Psychiatry and Behavioral Sciences, UMass Chan Medical School Elizabeth Thomas, Ph.D., Assistant Professor, Department of Social and Behavioral Sciences, Temple University College of Public Health Hilary Bertisch, Ph.D., ABPP, Assistant Professor, Zucker School of Medicine, Hofstra/Northwell Brittany Grimes, Project Manager of Youth and Young Adult Services, Oklahoma Department of Mental Health and Substance Abuse Services

Which Patients With HR+/HER2- EBC Benefit From Intensification of Therapy? Expert Recommendations for Clinical Assessment

https://www.medscape.org/viewarticle/which-patients-hr-her2-ebc-benefit-intensification-therapy-2025a10002qz?page=1&sso=true&uac=148436CN&src=mkmcmr_driv_stan_mscpedu_250529-OUS-HONC-which-patients-hr-her2-ebc-benefit-intensification-therapy-2025a10002qz-cta

AI and terrorism: How bioterrorists could easily fake an attack Jay Varma | STAT News | May 29, 2025

https://geneticliteracyproject.org/2025/05/29/ai-and-terrorism-how-bioterrorists-could-easily-fake-an-attack/

More than 300 people in the U.S. are still dying every week from COVID Mary Kekatos | Yahoo News | May 30, 2025

https://geneticliteracyproject.org/2025/05/30/more-than-300-people-in-the-u-s-are-still-dying-every-week-from-covid/

Viewpoint: Following no medical guidance, RFK, Jr. recklessly removes COVID vaccine recommendation for children and pregnant women Aimee Pugh Bernard, David Higgins, Elana Pearl BenJoseph, Jess Steier | Unbiased Science | May 29, 2025

https://geneticliteracyproject.org/2025/05/29/viewpoint-following-no-medical-guidance-rfk-jr-recklessly-removes-covid-vaccine-recommendation-for-children-and-pregnant-women/?mc_cid=861118dad3&mc_eid=b73600b7ac

Knocking down the walls that make pancreatic tumors unreachable An experimental drug significantly reduced tumor burden and extended survival in mice with pancreatic cancer.

https://www.drugdiscoverynews.com/knocking-down-the-walls-that-make-pancreatic-tumors-unreachable-15985?utm_campaign=DDN_Newsletter_Dose&utm_medium=email&_hsenc=p2ANqtz-8sAzVxIwkOumptY0bS5PHWo2VlbYbQtdG_-Yb2sM00EJphhz4jIuFIeuvqCaB_e4ocWYs7jby06pK6_Yce1Dg3TxBDjQ&_hsmi=363722390&utm_content=363722390&utm_source=hs_email

A dual-threat cancer drug takes aim at resistance mechanisms Judy Sebolt-Leopold and her team at MEKanistic Therapeutics developed a single molecule targeting two kinase enzymes that drive resistance to cancer therapies.

https://www.drugdiscoverynews.com/a-dual-threat-cancer-drug-takes-aim-at-resistance-mechanisms-16267?utm_campaign=DDN_Newsletter_Dose&utm_medium=email&_hsenc=p2ANqtz-_uFwgsT17e7YliZqlECrwj3aFbL8meTryu8sTGT8OBOY1qjBg2381ZmK22COctScUYJJ1nTGGFYhZlcZPWmgL7T0KMMw&_hsmi=363722390&utm_content=363722390&utm_source=hs_email

To fight HIV, antibodies boost the immune system HIV is incurable because it hides in latently infected cells. Researchers are hoping to change that — and prevent new infections — with the use of antibodies.

https://www.drugdiscoverynews.com/to-fight-hiv-antibodies-boost-the-immune-system-16406

New cancer fitness drugs aim to trigger tumor death Larkspur Biosciences scientists develop drugs that make colorectal cancer tumors vulnerable to killing by the immune system and cell death pathways.

https://www.drugdiscoverynews.com/new-cancer-fitness-drugs-aim-to-trigger-tumor-death-16407

Weekly Rundown: No COVID-19 vaccines for kids or pregnant women HHS decisions on COVID-19 and flu vaccines, new therapies with encouraging results for depression and ALS, and more industry news this week.

https://www.drugdiscoverynews.com/weekly-rundown-no-covid-19-vaccines-for-kids-or-pregnant-women-16414

GLP-1s: 3 Lesser-Known Downsides ++++++... American Association of Clinical Endocrinology (AACE) Annual Meeting 2025 May 15 - 17, 2025 | Orlando, Florida

Be Aware of Lesser-Known Downsides to GLP-1 Weight Loss Miriam E. Tucker May 22, 2025 https://www.medscape.com/viewarticle/be-aware-lesser-known-downsides-glp-1-weight-loss-2025a1000cwy?ecd=WNL_conf_endo_AACE-SPON_250529_MSCPEDIT_etid7455849&uac=148436CN&impID=7455849 Sotagliflozin Lowers A1c, Weight Even in Kidney Impairment Miriam E. Tucker May 17, 2025 https://www.medscape.com/viewarticle/sotagliflozin-lowers-a1c-weight-even-kidney-impairment-2025a1000ch7?ecd=WNL_conf_endo_AACE-SPON_250529_MSCPEDIT_etid7455849&uac=148436CN&impID=7455849 Fracture Risk Lower With Semaglutide vs Sleeve Gastrectomy Miriam E. Tucker May 16, 2025 https://www.medscape.com/viewarticle/fracture-risk-lower-semaglutide-vs-sleeve-2025a1000cf5?ecd=WNL_conf_endo_AACE-SPON_250529_MSCPEDIT_etid7455849&uac=148436CN&impID=7455849 Can Endocrine NP Training Help Ease Provider Shortage? Miriam E. Tucker May 27, 2025 https://www.medscape.com/viewarticle/can-endocrine-np-training-help-ease-provider-shortage-2025a1000d9w?ecd=WNL_conf_endo_AACE-SPON_250529_MSCPEDIT_etid7455849&uac=148436CN&impID=7455849 New Data Back Crinecerfont in Congenital Adrenal Hyperplasia Miriam E. Tucker May 15, 2025 https://www.medscape.com/viewarticle/new-data-back-crinecerfont-congenital-adrenal-hyperplasia-2025a1000cc7?ecd=WNL_conf_endo_AACE-SPON_250529_MSCPEDIT_etid7455849&uac=148436CN&impID=7455849 Palopegteriparatide Benefits Sustained in Hypoparathyroidism Miriam E. Tucker May 21, 2025 https://www.medscape.com/viewarticle/palopegteriparatide-benefits-sustained-hypoparathyroidism-2025a1000csw?ecd=WNL_conf_endo_AACE-SPON_250529_MSCPEDIT_etid7455849&uac=148436CN&impID=7455849 American Association of Clinical Endocrinology (AACE) Annual Meeting 2025 https://www.medscape.com/viewcollection/37797?ecd=WNL_conf_endo_AACE-SPON_250529_MSCPEDIT_etid7455849&uac=148436CN&impID=7455849

NIH scientists pioneer promising treatment for intractable cancer pain RTX is a non-addictive pain therapy derived from a cactus-like plant.

https://www.nih.gov/news-events/news-releases/nih-scientists-pioneer-promising-treatment-intractable-cancer-pain

Scientists identify diagnostic aid to determine risk of diabetic foot ulcer recurrence NIH-funded project shows that trans-epidermal water loss could indicate if wounds are fully healed. .

https://www.nih.gov/news-events/news-releases/scientists-identify-diagnostic-aid-determine-risk-diabetic-foot-ulcer-recurrence

Can Shorter RT Offer Long-Term Benefits in Prostate Cancer? Edited by Gargi Mukherjee May 30, 2025

https://www.medscape.com/viewarticle/can-shorter-rt-offer-long-term-benefits-prostate-cancer-2025a1000ejm

Do Stimulants Cause Psychosis in Kids? +++++ +++

A wake-up call: the second Commission on adolescent health +...

https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(25)00863-3/fulltext?dgcid=raven_jbs_etoc_email TheLancet.com will be undergoing maintenance on Saturday, May 31st from 8am to 12pm US Eastern (4 hours). During this timeframe, we ask that you refrain from logging in or registering to avoid any difficulty with this functionality. We apologise for the inconvenience. May 31, 2025 Volume 405Number 10493p1883-2022 https://www.thelancet.com/journals/lancet/issue/vol405no10493/PIIS0140-6736(25)X0022-2

Announcing The Lancet Obstetrics, Gynaecology, & Women’s Health +...

https://www.thelancet.com/obstetrics-gynaecology-womens-health?dgcid=raven_jbs_etoc_feature_lanogwlaunch TheLancet.com will be undergoing maintenance on Saturday, May 31st from 8am to 12pm US Eastern (4 hours). During this timeframe, we ask that you refrain from logging in or registering to avoid any difficulty with this functionality. We apologise for the inconvenience.

Most Read Papers - Cancer Science & Oncogenesis

https://read.qxmd.com/collection/1133?ecd=wnl_readmost_250530

Borderline Personality Disorder | Celiac Disease | Smoking Cessation | Bruxism | Memory Problems

https://content.govdelivery.com/accounts/USNIH/bulletins/3e28b76?reqfrom=share

jueves, 29 de mayo de 2025

Prospective evaluation of plasma proteins in relation to surgical endometriosis diagnosis in the Nurses’ Health Study II

https://www.thelancet.com/journals/ebiom/article/PIIS2352-3964(25)00132-X/fulltext?dgcid=hubspot_update_feature_updatealerts_ebiom&utm_campaign=update-ebiom&utm_medium=email&_hsenc=p2ANqtz--g43ub2WvGVQvlPxfcmhcUw7jZ4IKqqljBXqw6jRcF1myBa-TGQu-DmBPzlIUtr7TAvz4PA0BW7e2HJ5S643lmmoatKg&_hsmi=363701923&utm_content=363639642&utm_source=hs_email May 2025 Volume 115 https://www.thelancet.com/journals/ebiom/issue/vol115nonull/PIIS2352-3964(25)X0004-9

Effect of lithium on circadian activity level and flexibility in patients with bipolar disorder: results from the Oxford Lithium Trial

https://www.thelancet.com/journals/ebiom/article/PIIS2352-3964(25)00120-3/fulltext?dgcid=hubspot_update_feature_updatealerts_ebiom&utm_campaign=update-ebiom&utm_medium=email&_hsenc=p2ANqtz-8DO3cvhtqu3B3nQT8KTCpRm_fTnVfW_I8VAFyyiIXIwkOX2RAAwWJiyuGdrYTXHULLhnTv2oXjVLxKqQ3AivV1rTEUFQ&_hsmi=363701923&utm_content=363639642&utm_source=hs_email

Patients first: improving toxicity assessment and reporting +...

https://www.thelancet.com/journals/lanhae/article/PIIS2352-3026(25)00145-0/fulltext?dgcid=raven_jbs_etoc_email Jun 2025 Volume 12Number 6e401-e482 https://www.thelancet.com/journals/lanhae/issue/vol12no6/PIIS2352-3026(25)X0006-5

US Clean Air in the firing line +...

https://www.thelancet.com/journals/lanonc/article/PIIS1470-2045(25)00294-3/fulltext?dgcid=raven_jbs_etoc_email Issue cover for Volume 26, Issue 6 Jun 2025 Volume 26Number 6p663-816, e295-e331 https://www.thelancet.com/journals/lanonc/issue/vol26no6/PIIS1470-2045(25)X0006-1

Shaping the Future of Advanced Breast Cancer Care: A Deep Dive Into Novel Data and Emerging Therapies Authors: Volkmar Müller, MD; Judy King, MRCP, PhD

https://www.medscape.org/viewarticle/1002178?sso=true&uac=148436CN&src=mkmcmr_driv_stan_mscpedu_250528-OUS-HONC-1002178-cta

FDA Slams Pfizer for Talzenna Biomarker-Free Push M. Alexander Otto, PA, MMS May 29, 2025

https://www.medscape.com/viewarticle/fda-slams-pfizer-talzenna-biomarker-free-push-2025a1000e2u

Is 3D Mammography Better for Women With Family History of Breast Cancer? Edited by Gargi Mukherjee May 29, 2025

https://www.medscape.com/viewarticle/mammography-better-women-family-history-breast-cancer-2025a1000e0o American Society of Clinical Oncology (ASCO) 2025 https://www.medscape.com/viewcollection/37796

Cut the Clutter in Gynaecological Cancer Diagnosis Marcela Becegato May 29, 2025

https://www.medscape.com/viewarticle/cut-clutter-gynaecological-cancer-diagnosis-2025a1000e27

Defying Grim Prognosis to Celebrate a 20th ‘Cancerversary’ K. J. Bannan May 29, 2025

https://www.medscape.com/viewarticle/defying-grim-prognosis-celebrate-20th-cancerversary-2025a1000e5m

Can Popular Weight-Loss Drugs Protect Against Obesity-Related Cancers? Megan Brooks May 28, 2025

https://www.medscape.com/viewarticle/can-popular-weight-loss-drugs-protect-against-obesity-2025a1000dfl

The Latest Developments in Cancer Care and Office Management: Medscape Oncology Practice Issues Report 2025

https://www.medscape.com/slideshow/2025-oncology-practice-issues-6018243?ecd=WNL_physrep_250528_MSCPEDIT_info_etid7452908&uac=148436CN&impID=7452908

Complement-Mediated Kidney Disorders: A Case Series +...

https://checkrare.com/learning-center/courses/ Autoimmune/Auto-inflammatory Disorders https://checkrare.com/diseases/

Global Genes’ Patient and Advocate Initiatives By: Madaline Spencer| Published on: May 9, 2025 +++++ ++++

CLINICAL PERSPECTIVES Efficacy of Atrasentan To Treat Patients With IgA Nephropathy https://checkrare.com/efficacy-of-atrasentan-to-treat-patients-with-iga-nephropathy/ Richard Lafayette, MD, Professor of Medicine, Nephrology and Director of the Glomerular Disease Center at Stanford University Medical Center, discusses the recent approval of Vanrafia (atrasentan) for patients with IgA nephropathy (IgAN). New FDA-Approved C3 Glomerulopathy (C3G) Treatment Targets Underlying Cause of Disease https://checkrare.com/new-fda-approved-c3-glomerulopathy-c3g-treatment-targets-underlying-cause-of-disease/ Carla Nester, MD, Professor of Pediatrics-Nephrology at the University of Iowa, discusses the recent U.S. Food and Drug Administration (FDA) approval of iptacopan as the first and only treatment of adults with C3 glomerulopathy (C3G). Atypical Autosomal Dominant Polycystic Kidney Disease With IFT140 Variants https://checkrare.com/atypical-autosomal-dominant-polycystic-kidney-disease-with-ift140-variants/ This study evaluates the characterization of this atypical phenotype and focuses on 75 patients with ADPKD-like phenotypes with 41 different monoallelic predicted loss-of-function IFT140 variants. Navigating the Challenges of the Orphan Drug Market https://checkrare.com/navigating-the-challenges-of-the-orphan-drug-market/ Johanna Rossell, Senior Vice President and General Manager of Rare Diseases at Sumitomo Pharma America, discusses best practices for navigating challenges of the orphan drug market and provides personal experience through her work with congenital athymia. Mental Health and the Rare Disease Community https://checkrare.com/mental-health-and-the-rare-disease-community/ Al Freedman, PhD, Rare Disease Psychologist and Rare Dad, discusses how industry partners can support the mental health of rare disease communities. Challenges Faced by Rare Disease Patients in India https://checkrare.com/challenges-faced-by-rare-disease-patients-in-india/ Ramaiah Muthyala, PhD, Research Associate Professor at University of Minnesota and President and CEO of Indian Organization for Rare Diseases (IORD), discusses the challenges faced by rare disease patients in India. Global Genes’ Patient and Advocate Initiatives https://checkrare.com/global-genes-patient-and-advocate-initiatives/ Daniel DeFabio, Director of Community Engagement and Education at Global Genes, Co-Founder of Disorder: The Rare Disease Film Festival, and rare disease father, discusses Global Genes’ patient and advocate initiatives. Newborn Screening Update Following RFK’s Removal of Advisory Committee https://checkrare.com/newborn-screening-update-following-rfks-removal-of-advisory-committee/ Dean Suhr, President and co-founder of the MLD Foundation, discusses newborn screening updates and plans for gaining recommended uniform screening panel (RUSP) approval.

FDA Approves Inhalation Powder Formulation of Treprostinil for Pulmonary Arterial Hypertension

https://checkrare.com/

2025 Orphan Drugs: PDUFA Dates and FDA Approvals By: Peter Ciszewski| Updated on: May 15, 2025

https://checkrare.com/2025-orphan-drugs-pdufa-dates-and-fda-approvals/

Rare Disease Focus: Kidney and Urinary Diseases +++

LEARNING CENTER Optimizing Therapeutic Proteins Through PEGylation: Key Parameters and Impacts https://checkrare.com/optimizing-therapeutic-proteins-through-pegylation/ Professors João Gonçalves and Paolo Caliceti discuss how PEGylation represents a major technological advancement in the development and optimization of therapeutic proteins. Learn About WHIM Syndrome https://checkrare.com/learn-about-whim-syndrome/ WHIM syndrome is a rare, congenital primary immunodeficiency disorder associated with neutropenia that typically presents in childhood or adolescence, predominantly caused by pathogenic variants in the CXCR4 chemokine receptor gene. Fabry Disease https://checkrare.com/fabry-disease-podcast-series/ Fabry disease is a lysosomal storage disease characterized by insufficient alpha-galactosidase (alpha-GAL). Dr. William Burns, Dr. Nicola Longo, and Maya Kineen discuss diagnosis and personalized treatment plans.

International expert consensus statement on PICU admission and early critical care management for paediatric patients following haematopoietic cell transplant and immune effector cell therapy

https://www.thelancet.com/journals/lanchi/article/PIIS2352-4642(25)00091-4/abstract?utm_campaign=update-lanchi&utm_medium=email&dgcid=hubspot_update_feature_updatealerts_lanchi&_hsenc=p2ANqtz--2xK8F4-glUGDJSvX4Zo7TbSHu20cdJ-sxevAuvhtNneO7ccJTxtFUFw0q33gN1Q9NxnEloSlrtXcC-6o0wJoxsszSGQ&_hsmi=363655893&utm_content=363639589&utm_source=hs_email

Predicting paediatric pneumonia severity in the emergency department: a multinational prospective cohort study of the Pediatric Emergency Research Network

https://www.thelancet.com/journals/lanchi/article/PIIS2352-4642(25)00094-X/abstract?utm_campaign=update-lanchi&utm_medium=email&dgcid=hubspot_update_feature_updatealerts_lanchi&_hsenc=p2ANqtz---rZb8Pmqhiv9LANmjtrejRNbTc2RYK-qd-F8OAz59ycZfGjGxT-Vnlhkp9ufJKZd_UniPutZQpVA1PPBplEanjQMvzw&_hsmi=363655893&utm_content=363639589&utm_source=hs_email

Keeping families stable, secure, and together The Lancet Child & Adolescent Health

https://www.thelancet.com/journals/lanchi/issue/vol9no6/PIIS2352-4642(25)X0006-7

Insulin for early glycaemic abnormality in children with cystic fibrosis without cystic fibrosis-related diabetes (CF-IDEA): a randomised controlled trial

https://www.thelancet.com/journals/lanchi/article/PIIS2352-4642(25)00099-9/abstract?utm_campaign=update-lanchi&utm_medium=email&dgcid=hubspot_update_feature_updatealerts_lanchi&_hsenc=p2ANqtz-84rUIGfjNUVf0Hi1h4UnQnMmfbN7OHp3EC6JRvSRpirMynKUTZ9wsCgQUWBTfoUruPRdwwvFwV9Dw7Fj2bxfYY6yDYZA&_hsmi=363655893&utm_content=363639589&utm_source=hs_email

miércoles, 28 de mayo de 2025

Managing Adverse Events of Oral Adjuvant Therapies in Patients With HR+/HER2- Early Breast Cancer Authors: Joyce A. O’Shaughnessy, MD

https://www.medscape.org/viewarticle/managing-adverse-events-oral-adjuvant-therapies-patients-hr-2025a1000563?page=1&sso=true&uac=148436CN&src=mkmcmr_driv_stan_mscpedu_250527-OUS-HONC-managing-adverse-events-oral-adjuvant-therapies-patients-hr-2025a1000563-cta

European Congress on Obesity (ECO) 2025

https://www.medscape.com/viewcollection/37708?ecd=WNL_conf_endo_ECO-SPON_250527_MSCPEDIT_etid7444892&uac=148436CN&impID=7444892

European Congress on Obesity (ECO) 2025 May 11 - 14, 2025 | Málaga, Spain

Obesity App May Enable Weight Loss With Less Semaglutide Becky McCall, MSc, MScPh May 15, 2025 https://www.medscape.com/viewarticle/obesity-app-may-enable-weight-loss-less-semaglutide-2025a1000c0d?ecd=WNL_conf_endo_ECO-SPON_250527_MSCPEDIT_etid7444892&uac=148436CN&impID=7444892

ESMO Breast Cancer 2025 May 14 - 17, 2025 | Munich, Germany and Virtual ++++++

https://www.medscape.com/viewarticle/new-high-risk-triple-negative-breast-cancer-subgroup-2025a1000ci7?ecd=WNL_conf_onc_ESMO-NON-SPON_250527_MSCPEDIT_etid7446057&uac=148436CN&impID=7446057 Adjuvant Pertuzumab Shows OS Benefit in HER2 Breast Cancer Tara Haelle May 19, 2025 https://www.medscape.com/viewarticle/adjuvant-pertuzumab-shows-os-benefit-her2-breast-cancer-2025a1000cke?ecd=WNL_conf_onc_ESMO-NON-SPON_250527_MSCPEDIT_etid7446057&uac=148436CN&impID=7446057 Imlunestrant Benefit in Breast Cancer Holds After Progression on CDK4/6 Therapy Megan Brooks May 16, 2025 https://www.medscape.com/viewarticle/imlunestrant-benefit-breast-cancer-holds-after-progression-2025a1000cck?ecd=WNL_conf_onc_ESMO-NON-SPON_250527_MSCPEDIT_etid7446057&uac=148436CN&impID=7446057 Does Weight Gain After Breast Cancer Diagnosis Affect Outcomes? Megan Brooks May 20, 2025 https://www.medscape.com/viewarticle/does-weight-gain-after-breast-cancer-diagnosis-affect-2025a1000cp4?ecd=WNL_conf_onc_ESMO-NON-SPON_250527_MSCPEDIT_etid7446057&uac=148436CN&impID=7446057 Pregnancy Safe in Those Who Previously Had Breast Cancer While Pregnant Tara Haelle May 20, 2025 https://www.medscape.com/viewarticle/pregnancy-safe-those-who-previously-had-breast-cancer-while-2025a1000cpv?ecd=WNL_conf_onc_ESMO-NON-SPON_250527_MSCPEDIT_etid7446057&uac=148436CN&impID=7446057 https://www.medscape.com/viewcollection/37805?ecd=WNL_conf_onc_ESMO-NON-SPON_250527_MSCPEDIT_etid7446057&uac=148436CN&impID=7446057

COVID-19 and Oral Antivirals: How to Define an Eligible Patient Authors: Marta Boffito, MD, PhD, FRSP, MBA; Jörg Schelling, MD, FESPCH

https://www.medscape.org/viewarticle/1002037?sso=true&uac=148436CN&src=mkmcmr_driv_stan_mscpedu_250528-OUS-HONC-1002037-cta

CLINICAL PERSPECTIVES: CAMP4 Therapeutics’ Programs For Urea Cycle Disorders and SYNGAP1 +++++ +++++ +...

CLINICAL PERSPECTIVES CAMP4 Therapeutics’ Programs For Urea Cycle Disorders and SYNGAP1 https://checkrare.com/camp4-therapeutics-programs-for-urea-cycle-disorders-and-syngap1/ Yuri Maricich, MD, Chief Medical Officer of CAMP4 Therapeutics, discusses the company’s current drug development programs. A Patient’s Diagnostic Journey With Idiopathic Pulmonary Hemosiderosis (IPH) https://checkrare.com/a-patients-diagnostic-journey-with-idiopathic-pulmonary-hemosiderosis-iph/ David Curren, patient advocate and board member for Breath of Hope Rhode Island, discusses his grandson’s diagnostic journey with idiopathic pulmonary hemosiderosis. Navigating the Challenges of the Orphan Drug Market https://checkrare.com/navigating-the-challenges-of-the-orphan-drug-market/ Johanna Rossell, Senior Vice President and General Manager of Rare Diseases at Sumitomo Pharma America, discusses best practices for navigating challenges of the orphan drug market and provides personal experience through her work with congenital athymia. Mental Health and the Rare Disease Community https://checkrare.com/mental-health-and-the-rare-disease-community/ Al Freedman, PhD, Rare Disease Psychologist and Rare Dad, discusses how industry partners can support the mental health of rare disease communities. Urea Cycle Disorders: Overview and Developing Novel Therapies https://checkrare.com/urea-cycle-disorders-overview-and-developing-novel-therapies/ Yuri Maricich, MD, Chief Medical Officer of CAMP4 Therapeutics, provides an overview of urea cycle disorders (UCDs) and discusses developing novel therapies. Newly Approved Treatment Targets Underlying Cause of IgG4-Related Disease https://checkrare.com/newly-approved-treatment-targets-underlying-cause-of-igg4-related-disease/ Arezou Khosroshahi, MD, Associate Professor of Medicine at Emory University School of Medicine, discusses the approval of Uplizna (inebilizumab-cdon) for treatment of immunoglobulin G4-related disease (IgG4-RD). Positive Safety and Efficacy Data for Intrathecal Administration of Gene Therapy for SMA https://checkrare.com/positive-safety-and-efficacy-data-for-intrathecal-administration-of-gene-therapy-for-sma/ Norman Putzki, MD, Global Development Head of Neuroscience and Gene Therapy at Novartis, discusses positive safety and efficacy data for OAV101 IT, an investigational gene therapy for spinal muscular atrophy (SMA). Challenges Faced by Rare Disease Patients in India https://checkrare.com/challenges-faced-by-rare-disease-patients-in-india/ Ramaiah Muthyala, PhD, Research Associate Professor at University of Minnesota and President and CEO of Indian Organization for Rare Diseases (IORD), discusses the challenges faced by rare disease patients in India. Global Genes’ Patient and Advocate Initiatives https://checkrare.com/global-genes-patient-and-advocate-initiatives/ Daniel DeFabio, Director of Community Engagement and Education at Global Genes, Co-Founder of Disorder: The Rare Disease Film Festival, and rare disease father, discusses Global Genes’ patient and advocate initiatives. Ongoing Research for Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay https://checkrare.com/ongoing-research-for-autosomal-recessive-spastic-ataxia-of-charlevoix-saguenay/ Sonia Gobeil, co-founder of Ataxia of Charlevoix-Saguenay Foundation, discusses their organization and ongoing research for autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS). Newborn Screening Update Following RFK’s Removal of Advisory Committee https://checkrare.com/newborn-screening-update-following-rfks-removal-of-advisory-committee/ Dean Suhr, President and co-founder of the MLD Foundation, discusses newborn screening updates and plans for gaining recommended uniform screening panel (RUSP) approval. Recent Data Highlights Benefits of Sepiapterin in Patients With PKU https://checkrare.com/recent-data-highlights-benefits-of-sepiapterin-in-patients-with-pku/ Nicola Longo, MD, PhD, Professor and Chief of Division of Clinical Genetics at the University of California, Los Angeles, discusses data presented at the 2025 ACMG Annual Clinical Genetics Meeting highlighting the benefits of sepiapterin in patients with classical phenylketonuria (PKU). The Impact of Hemophilia on Women and Girls https://checkrare.com/the-impact-of-hemophilia-on-women-and-girls/ Shellye Horowitz, a patient with hemophilia and advocate, discusses the impact of hemophilia on women and girls. ATLG Versus ATG in Graft-Versus-Host Disease https://checkrare.com/atlg-versus-atg-in-graft-versus-host-disease/ A new study published in American Journal of Hematology compared two treatment options for Graft versus host disease (GVHD). Efficacy of Nipocalimab To Treat Myasthenia Gravis: Updates From Vivacity-MG3 Trial https://checkrare.com/efficacy-of-nipcalimab-to-treat-myasthenia-gravis-updates-from-vivacity-mg3-trial/ Sindhu Ramchandren, MD, Executive Medical Director, Neuroscience and Disease Cluster Lead for Neuroimmunology and Neuromuscular Disorders at Johnson & Johnson, discusses updated results from the Vivacity-MG3 clinical trial in patients with generalized myasthenia gravis (MG). Effect of Increased IgG Levels on Prognosis in Primary Biliary Cholangitis https://checkrare.com/effect-of-increased-igg-levels-on-prognosis-in-primary-biliary-cholangitis/ A recent study published in Liver International analyzed patients with primary biliary cholangitis (PBC) who presented with higher IgG levels at diagnosis. Safety and Efficacy of Zilucoplan to Treat Myasthenia Gravis: Results from the RAISE Trial https://checkrare.com/safety-and-efficacy-of-zilucoplan-to-treat-myasthenia-gravis-results-from-the-raise-trial/ Michael Weiss, MD, Department of Neurology, University of Washington Medical Center, discusses results from the RAISE clinical trial testing the safety and efficacy of zilucoplan in patients with myasthenia gravis (MG). Efficacy of Atrasentan To Treat Patients With IgA Nephropathy https://checkrare.com/efficacy-of-atrasentan-to-treat-patients-with-iga-nephropathy/ Richard Lafayette, MD, Professor of Medicine, Nephrology and Director of the Glomerular Disease Center at Stanford University Medical Center, discusses the recent approval of Vanrafia (atrasentan) for patients with IgA nephropathy (IgAN). Digital Voice Analysis as a Biomarker of Acromegaly https://checkrare.com/digital-voice-analysis-as-a-biomarker-of-acromegaly/ A recent study published in The Journal of Clinical Endocrinology & Metabolism analyzed the use of digital voice analysis as a biomarker of acromegaly. New FDA-Approved C3 Glomerulopathy (C3G) Treatment Targets Underlying Cause of Disease https://checkrare.com/new-fda-approved-c3-glomerulopathy-c3g-treatment-targets-underlying-cause-of-disease/ Carla Nester, MD, Professor of Pediatrics-Nephrology at the University of Iowa, discusses the recent U.S. Food and Drug Administration (FDA) approval of iptacopan as the first and only treatment of adults with C3 glomerulopathy (C3G). Clinical Outcomes in Peripheral Ulcerative Keratitis https://checkrare.com/clinical-outcomes-in-peripheral-ulcerative-keratitis/ A recent study published in the American Journal of Ophthalmology observed clinical outcomes in patients with peripheral ulcerative keratitis (PUK). FORWARD-53 Clinical Trial in Duchenne Muscular Dystrophy Shows Promise https://checkrare.com/forward-53-clinical-trial-in-duchenne-muscular-dystrophy-shows-promise/ Paul Bolno, MD, President and Chief Executive Officer of Wave Life Sciences, discusses positive results from the FORWARD-53 clinical trial evaluating WVE-N531 in patients with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. Outcomes of Relapsed or Refractory Diffuse Large B-Cell Lymphoma Treated With R-GemOx https://checkrare.com/outcomes-of-relapsed-or-refractory-diffuse-large-b-cell-lymphoma-treated-with-r-gemox/ A recent study evaluated outcomes of relapsed or refractory diffuse large B-cell lymphoma (DLBCL) patients treated with R-GemOx.

2025 Orphan Drugs: PDUFA Dates and FDA Approvals +++++

FDA NEWS 2025 Orphan Drugs: PDUFA Dates and FDA Approvals https://checkrare.com/2025-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 2025. FDA Approves Treatment for Pheochromocytoma or Paraganglioma https://checkrare.com/fda-approves-treatment-for-pheochromocytoma-or-paraganglioma/ The U.S. Food and Drug Administration (FDA) approved Welireg (belzutifan) for patients ages 12 years and older with locally advanced, unresectable or metastatic pheochromocytoma or paraganglioma. FDA Approves Myasthenia Gravis Treatment Nipocalimab https://checkrare.com/fda-approves-myasthenia-gravis-treatment-nipocalimab/ The U.S. FDA has approved Imaavy (nipocalimab) for the treatment of generalized myasthenia gravis (MG). The approval is for patients, 12 years of age and older, with anti-acetylcholine receptor (AChR) or anti-muscle-specific kinase (MuSK) positive antibodies. FDA Approves Gene Therapy for Epidermolysis Bullosa https://checkrare.com/fda-approves-gene-therapy-for-epidermolysis-bullosa-2/ The U.S. FDA has approved Zevaskyn (prademagene zamikeracel or pz-cel) for the treatment of wounds in patients with recessive dystrophic epidermolysis bullosa (EB). FDA Approves Hepatocellular Carcinoma Combination Therapy https://checkrare.com/fda-approves-hepatocellular-carcinoma-combination-therapy/ The U.S. FDA has approved nivolumab plus ipilimumab for first-line therapy in adult patients with unresectable or metastatic hepatocellular carcinoma (HCC).

Rare Disease News, Insight, and Clinical Perspective +++ +++ +++

FEATURED ARTICLES Progressive Familial Intrahepatic Cholestasis (PFIC): Diagnosing, Treating, Monitoring https://checkrare.com/progressive-familial-intrahepatic-cholestasis-pfic-diagnosing-treating-monitoring/ Patrick McKiernan, MD, Pediatric Hepatologist at Birmingham Children’s Hospital NHS Foundation Trust and Nadia Ovchinsky, MD, Professor of Medicine at NYU Grossman School of Medicine, discuss the recently published guidance on best practices to diagnose, treat, and monitor patients with progressive familial intrahepatic cholestasis (PFIC). Consider Rare: Suspecting and Diagnosing Fibrodysplasia Ossificans Progressiva (FOP) https://checkrare.com/suspecting-and-diagnosing-fibrodysplasia-ossificans-progressiva-fop/ Ellen Elias, MD, Professor, Pediatrics and Genetics, University of Colorado School of Medicine, and Christiaan Scott, MD, Professor of Medicine at the University of Ottawa, examine best practices to suspect and diagnose this ultra-rare condition. Neuroblastoma: Overview, Curie Scores, and Treatment Options https://checkrare.com/neuroblastoma-overview-curie-scores-and-treatment-options/ Greg Yanik, MD, Clinical Professor at University of Michigan’s C.S. Mott Children’s Hospital, provides an overview of this rare childhood cancer, goals of therapy, using the Curie score, and immotherapy treatments. Neuroblastoma: Evan Foundation https://checkrare.com/neuroblastoma-evan-foundation/ Gavin Lindberg details his family’s experience with neuroblastoma, provides advice to families, and discusses the EVAN Foundation. Improving Health Equity in Hereditary Angioedema (HAE): A Panel Discussion https://checkrare.com/improving-health-equity-in-hereditary-angioedema-hae-a-panel-discussion/ Aleena Banerji, MD, Timothy Craig, DO, and Marc Riedl, MD, provide an overview of the discrepancies in care observed in certain patient populations, as well as a discussion on best practices to reduce those inequalities moving forward. Optimizing Therapeutic Proteins Through PEGylation: Key Parameters and Impacts https://checkrare.com/optimizing-therapeutic-proteins-through-pegylation/ Professors João Gonçalves and Paolo Caliceti discuss how PEGylation represents a major technological advancement in the development and optimization of therapeutic proteins. Watch the DAYBUE® (trofinetide) in Practice Video Series to Hear Expert Insights on the First Treatment for This Rare Disease https://checkrare.com/watch-the-daybue-trofinetide-in-practice-video-series-to-hear-expert-insights/ Rett syndrome thought leaders discuss how DAYBUE® (trofinetide), the first and only FDA-approved treatment for Rett syndrome in adults and pediatric patients 2 years of age and older, plays a role in their patients’ treatment plans, while shedding light on their personal experiences with DAYBUE in their practices. Restless Legs Syndrome Treatment and Guidelines Update https://checkrare.com/restless-legs-syndrome-treatment-and-guidelines-update/ Mark J. Buchfuhrer, MD, discusses clinical practice guidelines update suggesting against the standard use of dopamine agonists for the treatment of restless legs syndrome (RLS).

Spinal cord stimulation: A new perspective for SMA research ++

https://rarerevolutionmagazine.com/spinal-cord-stimulation-a-new-perspective-for-sma-research/ To bring about a dramatic and wide reaching change in conditions and attitudes for the rare disease community. https://rarerevolutionmagazine.com/ The SMArt Horizon project is a collaborative initiative of SMA Europe and the SMA Foundation. Largely financially supported by FundAME through its Calendar Funds, (Spierziekten Nederland) and SMA Schweiz, it aims to identify innovative therapies for SMA, including exploring spinal cord stimulation (SCS) for people living with SMA. Seda Karakaya and Emilia Debska, of SMA Europe explain more Written by Seda Karakaya, Patient Advocate and SMA Europe Youth Group Member and Emilia Debska, Communications and Marketing Manager at SMA Europe Edited by Nicole Gusset, CEO, SMA Europe and Federica Fontana, Research and Medical Manager at SMA Europe At the forefront of this research is Dr. Marco Capogrosso, an accomplished researcher in SCS, who led the first in-human study investigating the use of epidural spinal cord stimulation to enhance function in people with SMA. His team’s innovative work is providing new insights into the potential of this therapy. Among the study’s participants is Carmen, who shares her experiences as one of the few people living with SMA to undergo this innovative potential treatment. Her journey offers a unique perspective on the study’s impact. Let’s explore the study’s findings and the important role of advocacy in advancing SMA research. Could you please say a few words about yourself? Dr. Marco Capogrosso: I was born in 1985 in Manduria, a town in Puglia, in the South of Italy. It’s a place known for its wine, stunning beaches, and great food. I have a Bachelor’s Degree in Physics, a Master’s Degree in Applied Physics from the University of Pisa, and a PhD in Biomedical Engineering from the Scuola Sant’Anna in Pisa. There, I worked on electrical stimulation of the nervous system and robotic systems. My journey in spinal cord stimulation began during my post-doctoral training at EPFL (École Polytechnique Fédérale de Lausanne) in Lausanne, Switzerland. I have a wide range of interests, from high-end cuisine, hiking, and sports like tennis and basketball to a deep love for science and culture. Most of all, I am passionate about my work as a scientist, which has been my greatest love since childhood. Carmen: “My name is Carmen. I’m 20 years old, and I’m from Madrid, Spain. I’m studying genetics, and I play wheelchair hockey and wheelchair football.” What inspired you to begin your research in the SMA field? Dr. Marco Capogrosso: Two key factors. First, the science itself. I read the work of Prof. George Mentis on the disease development and the underlying motor disorders. It just clicked in my head that the technology I was working on for spinal cord injury could actually be used even more effectively for SMA. Second, it was the mothers. I met so many mothers of children with SMA. Starting from Loren of the SMA Foundation to Mencia and the others at FundAME and SMA Europe. Man, if you want to change the world, nothing is more powerful than the strength of a mother trying to help her child. I just felt humbled in front of such strength. I couldn’t do it. I could not be that strong. But they are incredible. Just incredible. I look at my children and I hope I could be just half as strong as these mums. So, these mums, what did they do? They charged me with my responsibility. This isn’t the same game that I played when I was five to match the dinosaur bone. These are their children. I can help them. So, I have the responsibility to do it. And to do it seriously. And this understanding changed my career. What inspired you to pursue epidural spinal cord stimulation research, and how did you become involved in the SMArt Horizon project? Dr. Marco Capogrosso: I started working in spinal cord stimulation because when I was about to finish my PhD, the first works showing that SCS may work to improve motor control.And I was shocked that nobody knew how it worked. I figured that I had the right knowledge to work on that because I was a physicist, and I had worked on my whole PhD with Silvestro Micera on electrical stimulation of neurons. Here was a pristine scientific field that could make a huge clinical impact. Therefore, I throw myself at the challenge, and I think I contributed significantly to the theory of how SCS improves motor neuron function in the human spinal cord. Like I said above, once I knew this, I just had to connect the dots between what I knew and the research in SMA. I was then encouraged to explore the field because they realised that we needed new assistive technologies now that SMN (survival motor neuron) therapies are available. So here I am. How did you first hear about the SMArt Horizon study, and what motivated you to participate? What were your initial thoughts when you learned about spinal cord stimulation as a potential treatment for SMA? Carmen: The first time I heard about this project was in October 2024, at the conference organised by FundAME (SMA family foundation in Spain). In this conference, Genis Prat-Ortega, one of the researchers, came to talk to us about this project, and the truth is that I found it very interesting since I had never heard anything like this when it comes to a potential new treatment for SMA. My first thoughts when I found out about this study and the results it was giving were that I thought it was amazing to see someone with SMA improving and gaining strength so quickly. This had never been possible before. As a researcher, what has been the most rewarding aspect of working on this first-in-human study? Dr. Marco Capogrosso: Again, two things: First, the patients. What incredible people! I know people think that I say this because I should, right? But no, I mean it. What incredible people! Every one of them, intelligent, motivated, happy. An incredibly rewarding human experience. You certainly know that many people think of people with disability with pity. They are sad for them. But interacting with our participants made me happier, healthier at a fundamental human level. Significantly enriched my emotional story. These people are just incredible. This community, wow. I don’t know why that is, maybe you have that answer. Second is the results. I have never in my life had an experiment more successful and replicable than this one. And coupling that with the smile of our patients as they work with our team made the story of my career. If spinal cord stimulation becomes more widely accessible, what specific aspects of daily life do you think it could impact the most for people who live with SMA? Carmen: I think this study can help many people with SMA, since there are very simple things that we can’t do because, over the years, we have been losing our strength. Thanks to this therapy, I think we can recover some of the day-to-day skills that we have lost. These things can be actions as simple as opening doors, taking objects from shelves… I think this could help people who can’t perform actions, such as eating or brushing their teeth, to do it independently. This is a great advance, since it means that we can be more autonomous in our everyday lives. Looking ahead, what are the biggest challenges and opportunities in bringing spinal cord stimulation from research to real-world application for individuals with SMA? Dr. Marco Capogrosso: There are enormous regulatory and business challenges. Convincing companies that it is worth pursuing this, that somebody will make money out of it. It’s frustrating and very hard. But Gregoire (my post-doc mentor) once told me: “A problem shows its worth by fighting back”. And we will fight. What did you like the most about this adventure, apart from participating in the study itself? Carmen: It has been a great experience.Even though I had to leave everything I was doing in Spain to go to the United States for seven weeks, it has been incredible. Being able to be there and meet another culture, improve my English and meet other people, like all the team I have been with all this time, has been incredible. I’m very, very grateful for all their work and what is being achieved with this trial. I’m convinced that their work will change many lives. That’s why I hope this potential new treatment reaches many more people with SMA soon. What did you like the most about working with Carmen and her mother? Marco Capogrosso: They brought the sun to Pittsburgh during the darkest and coldest period of the year. When I think back to February, the first thing that comes to mind is their smile and the smiles of my incredible team members, Serena Donadio, Genis Prat-Ortega, Scott Ensel, and Julia Ostrowski. What role do you think patient organisations may play in future research projects focusing on SMA? Marco Capogrosso: Increasing awareness in the patient population and clinicians—and potentially putting pressure on regulatory bodies and large companies to support the development of medical devices for small but underserved patient populations. What message would you like to share with the SMA community about the importance of patient involvement in studies like this? Carmen: As a patient, I think it is very important that we get involved in these types of studies. If we want more treatments to be found for SMA and, one day, hopefully, to have a cure, people who live with SMA must participate. In my case, it is the second study in which I participate, and I think it is very important that people participate, because… if we don’t, who will?

Finding Comfort in Community: 20+ Support Groups that Help Patients and Families +++++

https://getpalliativecare.org/finding-comfort-in-community-20-support-groups-that-help-patients-and-families-25/?utm_source=Get+Palliative+Care&utm_campaign=e0ae343bce-GPC+-+Support+Groups+%2F+05.28.25&utm_medium=email&utm_term=0_4ef32a103e-e0ae343bce-378264050 How to Manage Anxiety and Depression When Living with a Serious Illness https://getpalliativecare.org/how-to-manage-anxiety-and-depression-when-living-with-a-serious-illness2022/?utm_source=Get+Palliative+Care&utm_campaign=e0ae343bce-GPC+-+Support+Groups+%2F+05.28.25&utm_medium=email&utm_term=0_4ef32a103e-e0ae343bce-378264050 7 Ways to Improve Your Sleep When Living with a Serious Illness https://getpalliativecare.org/7-ways-to-improve-your-sleep-when-living-with-a-serious-illness/?utm_source=Get+Palliative+Care&utm_campaign=e0ae343bce-GPC+-+Support+Groups+%2F+05.28.25&utm_medium=email&utm_term=0_4ef32a103e-e0ae343bce-378264050 More Patient Stories: Palliative Care Helps with Stress, Support, and More https://getpalliativecare.org/more-patient-stories-palliative-care-helps-with-stress-support-and-more/?utm_source=Get+Palliative+Care&utm_campaign=e0ae343bce-GPC+-+Support+Groups+%2F+05.28.25&utm_medium=email&utm_term=0_4ef32a103e-e0ae343bce-378264050 On-Demand Video: How Palliative Care Can Help People Living with Parkinson’s Disease https://getpalliativecare.org/

Muscle-loss concerns lead to new drug discovery platform ProFuse’s novel technology addresses the growing need to preserve muscle in the face of aging and the popularity of weight-loss drugs.

https://www.israel21c.org/muscle-loss-concerns-lead-to-new-drug-discovery-platform/

Song lyrics help people cope with trauma Study reveals that people often turn to songs with words that reflect their mental struggles to regulate their emotional state.

https://www.israel21c.org/song-lyrics-help-people-cope-with-trauma/

Prenatal stress impacts babies, especially girls, later on Study finds that even before babies take their first breath, the stress their mothers experience can shape how their own bodies will manage stress.

https://www.israel21c.org/prenatal-stress-impacts-babies-especially-girls-later-on/

Noncontact radar unit monitors patients’ vitals remotely Israeli lab develops system that tracks vital signs of multiple patients at once, without wires and without exposing medical staff to pathogens.

https://www.israel21c.org/noncontact-radar-unit-monitors-patients-vitals-remotely/

War woes kickstart surge in medical innovation Since war broke out with Hamas, Israel has faced an unprecedented health crisis, sparking an upsurge in rehab innovation that promises to improve healthcare worldwide.

https://www.israel21c.org/war-woes-kickstart-surge-in-medical-innovation/

Hemorrhage survival tripled in study by Israeli researchers Trauma treatment developed by Hebrew University and IDF Medical Corps has boosted survival from 25% to 73% in a severe blood loss model.

https://www.israel21c.org/hemorrhage-survival-tripled-in-study-by-israeli-researchers/

Origami vaccines fold up to fight cancer As self-assembling structures, DNA and RNA origami vaccines hold vaccine components in precise numbers and arrangements to help the immune system attack tumors.

https://www.drugdiscoverynews.com/origami-vaccines-fold-up-to-fight-cancer-15627?utm_campaign=DDN_Newsletter_Dose&utm_medium=email&_hsenc=p2ANqtz--ezEeb7LnMgeu7X3mKuu3uITjkD7ajC6ntI-OpjBqz2bq4bFytsBw5DELaP6wiGtrZLuuN0QWnGLZyySRq2PO9mdphVA&_hsmi=363434868&utm_content=363434868&utm_source=hs_email

Customizing mRNA vaccines for cancer Researchers use mRNA to create cancer vaccines that are personalized to each individual’s unique tumor.

https://www.drugdiscoverynews.com/customizing-mrna-vaccines-for-cancer-15894?utm_campaign=DDN_Newsletter_Dose&utm_medium=email&_hsenc=p2ANqtz-9TTbLiuvpn6f2ILCHseWTfrAgoiKcModT1mZJmOH-MEdxsDQdCekoGIgZqmcyTxHhzenTXhNvMFHy8KcpxYN0REBabRA&_hsmi=363434868&utm_content=363434868&utm_source=hs_email

Are you struggling with macrophage differentiation from induced pluripotent stem cells (iPSCs)?

https://go2.lonza.com/lbs-X-VIVO-research-applications.html?utm_campaign=DDN_Newsletter_Dose&utm_medium=email&_hsenc=p2ANqtz-8BDPaFH1r6-CzOeyyUqaISCeI4dsIf6J639jM9_iO5el4FQDjb5i5uICLCux9xLsdKJaYUehFz4Hw7XqnKfUzx3zIaNQ&_hsmi=363434868&utm_content=363434868&utm_source=hs_email

Targeting solid tumors two ways with one cancer vaccine IO Biotech’s two-pronged cancer vaccine targets tumors and the suppressive immune cells around them, demonstrating promising results in metastatic melanoma.

https://www.drugdiscoverynews.com/targeting-solid-tumors-two-ways-with-one-cancer-vaccine-16397

Could residues of growth promoters used in meat production result in Olympic athletes testing positive for drug use? UCLA Health Sciences | Food Manufacturing | May 27, 2025

https://geneticliteracyproject.org/2025/05/27/could-residues-of-growth-promoters-used-in-meat-production-result-in-olympic-athletes-testing-positive-for-drug-use/

The science of sitting—and why walking may be better for our bodies than running Brian Foster | Glass Almanac | May 27, 2025

https://geneticliteracyproject.org/2025/05/27/the-science-of-sitting-and-why-walking-may-be-better-for-our-bodies-than-running/

On chronic disease, the Make America Healthy Commissions misfires, but there are solutions: Rethinking fruits and vegetables Kevin Folta | May 27, 2025

https://geneticliteracyproject.org/2025/05/27/on-chronic-disease-the-make-america-healthy-commissions-misfires-but-there-are-solutions-rethinking-fruits-and-vegetables/

Longevity seekers misunderstand a fundamental truth about biology You can’t program the body like a computer

https://www.statnews.com/2025/05/27/biohackers-longevity-continuous-glucose-monitoring-programming-body/

Participant in Rocket Pharmaceuticals gene therapy study dies The company did not disclose the age of the person, who had enrolled in the study of patients with a condition called Danon disease

https://www.statnews.com/2025/05/27/rocket-pharmaceuticals-danon-disease-gene-therapy-patient-death/

At a major genome editing summit, spotlight turns to the value of human life Committee calls for charter on technologies like CRISPR that protects human dignity Manage alerts for this article Email this articleAt a major genome editing summit, spotlight turns to the value of human life Committee calls for charter on technologies like CRISPR that protects human dignity

https://www.statnews.com/2025/05/27/global-observatory-genome-editing-summit-crispr-ethics-human-dignity/

Clinical Cases in HR-Positive, HER2-Negative Early Breast Cancer: Applying the Latest Evidence Authors: William J. Gradishar, MD; Aditya Bardia, MD, MPH, FASCO; Seth Wander, MD, PhD

https://www.medscape.org/viewarticle/1002256?sso=true&uac=148436CN&src=mkmcmr_driv_stan_mscpedu_250526-OUS-HONC-1002256-cta

A Year in Review of Antibody-Drug Conjugates for Breast Cancer: Trends, Practice, and Outlook Authors: Peter Schmid, MD, PhD, FRCP; Shanu Modi, MD; Erika P. Hamilton, MD; Nicola Fusco, MD

https://www.medscape.org/viewarticle/1002233?sso=true&uac=148436CN&src=mkmcmr_driv_stan_mscpedu_250527-OUS-HONC-1002233-cta

A Ministroke Can Have Major Consequences By Paula Span May 27, 2025

https://kffhealthnews.org/news/article/ministroke-transient-ischemic-attack-tia-major-consequences/

lunes, 26 de mayo de 2025

Featured Article: Validation of an web-based dietary assessment tool (RiksmatenFlex) against doubly labelled water and 24 h dietary recalls in pregnant women +++

https://nutritionj.biomedcentral.com/ Nutrition & Metabolism https://nutritionandmetabolism.biomedcentral.com/ Journal of Health, Population and Nutrition https://jhpn.biomedcentral.com/

Effectiveness of non-invasive brain stimulation in improving sleep quality in insomnia: a systematic review

https://sleep.biomedcentral.com/

Association between headaches and lifestyle factors and physical and mental symptoms among 63,071 workers at a Japanese information technology company

https://thejournalofheadacheandpain.biomedcentral.com/

Orphanet Journal of Rare Diseases

https://ojrd.biomedcentral.com/?utm_source=bmc_etoc&utm_medium=email&utm_campaign=CONR_13023_AWA1_GL_DTEC_054CI_TOC-250525

Billy Joel diagnosed with brain condition — what to know about his treatment options Normal pressure hydrocephalus causes fluid buildup in the brain, often mimicking dementia Melissa Rudy By Melissa Rudy Fox News Published May 24, 2025 3:37pm EDT

Billy Joel diagnosed with brain condition — what to know about his treatment options Normal pressure hydrocephalus causes fluid buildup in the brain, often mimicking dementia Melissa Rudy By Melissa Rudy Fox News Published May 24, 2025 3:37pm EDT https://www.foxnews.com/health/billy-joel-diagnosed-brain-condition-heres-what-know-about-his-diagnosis

Study directly compares Zepbound and Wegovy for weight-loss results Researchers find tirzepatide leads to greater reduction in body weight and waist size Melissa Rudy By Melissa Rudy Fox News Published May 26, 2025 5:00am EDT

Study directly compares Zepbound and Wegovy for weight-loss results Researchers find tirzepatide leads to greater reduction in body weight and waist size Melissa Rudy By Melissa Rudy Fox News Published May 26, 2025 5:00am EDT https://www.foxnews.com/health/study-compares-zepbound-wegovy-weight-loss-direct-comparison