domingo, 31 de agosto de 2025
Insights into clinical features and genetic variants of Cornelia de Lange syndrome in China
https://www.news-medical.net/news/20250829/Insights-into-clinical-features-and-genetic-variants-of-Cornelia-de-Lange-syndrome-in-China.aspx
Cornelia de Lange syndrome (CdLS) is a rare genetic disorder with symptoms, including facial anomalies (such as fused eyebrows, short nose, upturned nose tip, and downturned mouth corners), growth retardation, small head (microcephaly), developmental delays, cognitive impairments, excess hair growth, and limb anomalies. Currently, mutations in seven genes (NIBPL, SMC1A, SMC3, RAD21, BRD4, HDAC8, and ANKRD11) are known to be associated with CdLS. CdLS is diagnosed based on both genetic testing and clinical examination. However, the clinical and genetic characteristics of CdLS in the Chinese population have not been determined.
First ESC statement on mental health and heart disease promotes integrated care
https://www.news-medical.net/news/20250830/First-ESC-statement-on-mental-health-and-heart-disease-promotes-integrated-care.aspx
A new ESC Clinical Consensus Statement is calling for greater awareness of the multidirectional relationship between mental health conditions and cardiovascular disease to improve patient health. The first ever ESC Clinical Consensus Statement to be developed on this topic was published today at ESC Congress 2025.
Updated guidelines aim to standardize valvular heart disease care across Europe
https://www.news-medical.net/news/20250830/Updated-guidelines-aim-to-standardize-valvular-heart-disease-care-across-Europe.aspx
Updated guidelines aim to standardize valvular heart disease care across Europe
Updated ESC/EACTS Guidelines, being published today at ESC Congress 2025, aim to improve the way that patients with valvular heart disease are diagnosed and treated, responding to robust new evidence that suggests some newer and less invasive treatments could be offered more widely and consistently to patients.
New ESC guidelines introduce unified approach to improve myocarditis and pericarditis care
https://www.news-medical.net/news/20250830/New-ESC-guidelines-introduce-unified-approach-to-improve-myocarditis-and-pericarditis-care.aspx
New ESC guidelines introduce unified approach to improve myocarditis and pericarditis care
New ESC Guidelines to improve the diagnosis and care of patients with myocarditis and pericarditis, have been published today at ESC Congress 2025.
ESC releases new guidance on lipid management to reduce cardiovascular risk
ESC releases new guidance on lipid management to reduce cardiovascular risk
New ESC/EAS Focused Update to Guidelines, about how best to manage lipid levels in patients to reduce cardiovascular risk, recommends the use of new cardiovascular risk predication algorithms. The update was published today at ESC Congress 2025.
https://www.news-medical.net/news/20250830/ESC-releases-new-guidance-on-lipid-management-to-reduce-cardiovascular-risk.aspx
New ESC guidelines prioritize women's autonomy in high-risk pregnancies
New ESC guidelines prioritize women's autonomy in high-risk pregnancies
Updated ESC Guidelines, published today at ESC Congress 2025, put a focus on a woman's autonomy in making her own reproductive choices by promoting a transparent dialogue and shared decision-making for pregnancies that are high-risk for an adverse maternal and/or fetal event.
https://www.news-medical.net/news/20250830/New-ESC-guidelines-prioritize-womens-autonomy-in-high-risk-pregnancies.aspx
sábado, 30 de agosto de 2025
Ultra-processed foods undermine diet quality in Mediterranean youth, study shows
https://www.news-medical.net/news/20250818/Ultra-processed-foods-undermine-diet-quality-in-Mediterranean-youth-study-shows.aspx?utm_source=news_medical_newsletter&utm_medium=email&utm_campaign=children_s_health_newsletter_26_august_2025
Evaluating Nerandomilast in Patients With Rare Pulmonary Fibrotic Conditions ++++++
CLINICAL PERSPECTIVES
Evaluating Nerandomilast in Patients With Rare Pulmonary Fibrotic Conditions
https://checkrare.com/evaluating-nerandomilast-in-patients-with-rare-pulmonary-fibrotic-conditions/
Leticia Orsatti, MD, Vice President of Clinical Development and Medical Affairs at Boehringer Ingelheim, discusses results from clinical trials examining the safety and efficacy of nerandomilast to treat patients with idiopathic pulmonary fibrosis (IPF) or progressive pulmonary fibrosis (PPF).
Diagnostic Odyssey With Histiocytosis
https://checkrare.com/diagnostic-odyssey-with-histiocytosis/
Nate Milam II, Patient Advocate for the Histiocytosis Association, discusses his diagnostic odyssey with histiocytosis.
Evolving Policy Landscapes for Rare Disease Access
https://checkrare.com/evolving-policy-landscapes-for-rare-disease-access/
Deb Jennings, Head of North America Patient Services Operations at Kyowa Kirin, discusses evolving policy landscapes for rare disease access.
The Role of Mental Health in Rare Disease Patient Outcomes
https://checkrare.com/the-role-of-mental-health-in-rare-disease-patient-outcomes/
Sumira Riaz, PhD, Health Psychologist & Patient Engagement Consultant at Unboxed Psychology, discusses the role of mental health in rare disease patient outcomes.
The Undiagnosed Disease Network
https://checkrare.com/the-undiagnosed-disease-network/
Kimberly LeBlanc, Genetic Counselor, Director of the Undiagnosed Diseases Network (UDN) Coordinating Center at Harvard Medical School, discusses approaching variants of uncertain significance in rare diseases.
Ethical Concerns in Rare Diseases and Expanded Access Programs
https://checkrare.com/ethical-concerns-in-rare-diseases-and-expanded-access-programs/
Alison Bateman-House, PhD, Assistant Professor Division of Medical Ethics at NYU Grossman School of Medicine, discusses ethical concerns in rare diseases and expanded access programs.
viernes, 29 de agosto de 2025
Editorial Moving care into the community The Lancet Primary Care + +...
Active case detection using loop-mediated isothermal amplification technology and treatment versus standard of care for malaria in pregnancy in Ethiopia (LAMPREG): a pragmatic randomised diagnostic outcomes trial
https://www.thelancet.com/journals/lanprc/article/PIIS3050-5143(25)00008-1/fulltext?dgcid=raven_jbs_etoc_feature_lanprc
Editorial
Moving care into the community
The Lancet Primary Care
https://www.thelancet.com/journals/lanprc/issue/vol1no2/PIIS3050-5143(25)X0003-0
Editorial p885 The dialogue around NCDs needs to change The Lancet + +...
Blood pressure-lowering efficacy of antihypertensive drugs and their combinations: a systematic review and meta-analysis of randomised, double-blind, placebo-controlled trials
https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(25)00991-2/abstract?dgcid=raven_jbs_etoc_feature_lancet
Editorial
p885
The dialogue around NCDs needs to change
The Lancet
https://www.thelancet.com/journals/lancet/issue/vol406no10506/PIIS0140-6736(25)X0035-0
SPEECH STRUGGLE: WHAT CAUSES STUTTERING?
Speech Struggle: What Causes Stuttering?
By Ivanhoe Broadcast News on September 1, 2025
https://www.ivanhoe.com/?p=35953&preview=1&_ppp=aae307d946
HEARTBREAK AND HEALTH: KNOW YOUR BROKEN HEART
Heartbreak And Health: Know Your Broken Heart
By Ivanhoe Broadcast News on September 2, 2025
https://www.ivanhoe.com/?p=35956&preview=1&_ppp=6d8384144e
REDUCING KNEE PAIN WITHOUT SURGERY
Reducing Knee Pain Without Surgery
By Ivanhoe Broadcast News on September 3, 2025
https://www.ivanhoe.com/?p=35961&preview=1&_ppp=2439488e96
ROBOTIC HYSTERECTOMIES: 3D BETTER THAN 2D
Robotic Hysterectomies: 3D Better Than 2D
By Ivanhoe Broadcast News on September 4, 2025
https://www.ivanhoe.com/?p=35964&preview=1&_ppp=ae9d860e12
WAYS TO MANAGE BLOOD SUGAR
Ways To Manage Blood Sugar
By Ivanhoe Broadcast News on September 5, 2025
https://www.ivanhoe.com/?p=35967&preview=1&_ppp=5b1cb64677
jueves, 28 de agosto de 2025
Editorial Diphtheria's resurgence and the high cost of misinformation eBioMedicine + +... +
A bedside-to-bench translational analysis of NF1 alterations and CDK4/6 inhibitor resistance in hormone receptor-positive metastatic breast cancer
https://www.thelancet.com/journals/ebiom/article/PIIS2352-3964(25)00272-5/fulltext?dgcid=hubspot_update_feature_updatealerts_ebiom&utm_campaign=update-ebiom&utm_medium=email&_hsenc=p2ANqtz-9c2YFMmu_hNakhQCqv-QF49xQhwQ5uXUs-dvBGHOxnK6bNWmsZycNvNRVqlBrMgXr_4YRgqYCRDqvsK58Xe0LrxQ9SGw&_hsmi=377845293&utm_content=377569936&utm_source=hs_email
Editorial
Diphtheria's resurgence and the high cost of misinformation
eBioMedicine
https://www.thelancet.com/journals/ebiom/issue/vol118nonull/PIIS2352-3964(25)X0007-4
Integrative immune analysis in patients with leprosy reveals host factors associated with mycobacterial control
https://www.thelancet.com/journals/ebiom/article/PIIS2352-3964(25)00299-3/fulltext?dgcid=hubspot_update_feature_updatealerts_ebiom&utm_campaign=update-ebiom&utm_medium=email&_hsenc=p2ANqtz-8-WX9edVqPoFbe7eYjCb8Pr7TYlZX9uKr1EYsr1lPwOHqrS0RSBdgfipWR585id2h9nJU6JKYv1wdiIN2fSgzJNDvd-w&_hsmi=377845293&utm_content=377569936&utm_source=hs_email
Editorial p619 Greater than its parts The Lancet Psychiatry ++... +
Holistic prevention and management of physical health side-effects of psychotropic medication: second report of the Lancet Psychiatry Physical Health Commission
https://www.thelancet.com/journals/lanpsy/article/PIIS2215-0366(25)00162-2/abstract?utm_campaign=update-lanpsy&utm_medium=email&dgcid=hubspot_update_feature_updatealerts_lanpsyphysicalhealth25&_hsenc=p2ANqtz-8ufXNdVOx2yalcylMNoBMtaa14wmArgkDJe84xU5NnTStzP_UwFg1JKK9hwmVlmH8Iz3qHXosBmrIn4ssbLO3hX3nn6Q&_hsmi=377839098&utm_content=377083434&utm_source=hs_email
Editorial
p619
Greater than its parts
The Lancet Psychiatry
https://www.thelancet.com/journals/lanpsy/issue/vol12no9/PIIS2215-0366(25)X0009-2
Implementing lifestyle interventions in mental health care: third report of the Lancet Psychiatry Physical Health Commission
https://www.thelancet.com/journals/lanpsy/article/PIIS2215-0366(25)00170-1/abstract?utm_campaign=update-lanpsy&utm_medium=email&dgcid=hubspot_update_feature_updatealerts_lanpsyphysicalhealth25&_hsenc=p2ANqtz-94DoavYIFLhtbpYkGsdvlgHVGkHtCfwJo00OLwAMvinzmAw4K8TLNSX8L8fy68Zeb2Lphq-AoCrfsHXiYunt5RopjRng&_hsmi=377839098&utm_content=377083434&utm_source=hs_email
Editorial e603 A moment of reckoning The Lancet HIV +++...
Bictegravir, emtricitabine, and tenofovir alafenamide versus ritonavir-boosted protease inhibitor-based antiretroviral therapy in people with HIV and viral suppression on second-line therapy in Haiti: an open-label, randomised, non-inferiority trial
Read this Article
https://www.thelancet.com/journals/lanhiv/article/PIIS2352-3018(25)00130-4/abstract?dgcid=raven_jbs_etoc_feature_lanhiv
The clinical and economic impact of genotypic resistance testing for people diagnosed with persistent virological non-suppression on tenofovir–lamivudine–dolutegravir in South Africa: a modelling study
Read this Article
https://www.thelancet.com/journals/lanhiv/article/PIIS2352-3018(25)00164-X/abstract?dgcid=raven_jbs_etoc_feature_lanhiv
Editorial
e603
A moment of reckoning
The Lancet HIV
https://www.thelancet.com/journals/lanhiv/issue/vol12no9/PIIS2352-3018(25)X0009-6
Editorial e722 Tackling health inequalities in Indigenous Peoples The Lancet Public Health +++
National, regional, and global statistics on alcohol consumption and associated burden of disease 2000–20: a modelling study and comparative risk assessment
https://www.thelancet.com/journals/lanpub/article/PIIS2468-2667(25)00174-4/fulltext?dgcid=raven_jbs_etoc_feature_lanpub
Editorial
e722
Tackling health inequalities in Indigenous Peoples
The Lancet Public Health
https://www.thelancet.com/journals/lanpub/issue/vol10no9/PIIS2468-2667(25)X0009-8
Alcohol's health burden: secondhand harms matter
https://www.thelancet.com/journals/lanpub/article/PIIS2468-2667(25)00198-7/fulltext?dgcid=raven_jbs_etoc_email
miércoles, 27 de agosto de 2025
Can eating yogurt every week lower your type 2 diabetes risk?
https://www.news-medical.net/news/20250826/Can-eating-yogurt-every-week-lower-your-type-2-diabetes-risk.aspx
With type 2 diabetes rates soaring, new FDA-approved messaging highlights yogurt as a practical dietary choice backed by observational evidence linking regular intake to reduced disease risk.
Do GLP-1 drugs shrink your muscles? New study explains the risks and what to do
https://www.news-medical.net/news/20250826/Do-GLP-1-drugs-shrink-your-muscles-New-study-explains-the-risks-and-what-to-do.aspx
Weight-loss drugs like semaglutide may melt fat, but at the cost of muscle mass. A new review reveals how to protect your strength while reaping the benefits.
Who eats the most pulses in America, and why it matters for health
https://www.news-medical.net/news/20250827/Who-eats-the-most-pulses-in-America-and-why-it-matters-for-health.aspx
Despite their low cost and health benefits, pulses remain under-consumed in the U.S., but new data reveal who eats them, why they are essential for health, and how small changes could close the gap with national dietary goals.
Why Salmonella Dublin poses a food safety threat in beef and dairy
https://www.news-medical.net/news/20250827/Why-Salmonella-Dublin-poses-a-food-safety-threat-in-beef-and-dairy.aspx
A nationwide genomic study shows that while Salmonella Dublin looks genetically uniform, it hides powerful resistance traits that threaten cattle, people, and the food supply alike.
Eating more animal protein may slightly lower cancer mortality
https://www.news-medical.net/news/20250827/Eating-more-animal-protein-may-slightly-lower-cancer-mortality.aspx
New findings challenge the long-held belief that animal protein raises mortality risk, showing no harm for longevity and even a small cancer-protective effect in US adults.
FDA removes risk evaluation and mitigation strategy (REMS) program for the antipsychotic drug Clozapine
https://www.fda.gov/drugs/drug-safety-and-availability/fda-removes-risk-evaluation-and-mitigation-strategy-rems-program-antipsychotic-drug-clozapine?utm_medium=email&utm_source=govdelivery
FDA Removes Risk Evaluation and Mitigation Strategies (REMS) Program for the Antipsychotic Drug Clozapine
The U.S. Food and Drug Administration (FDA) removed the risk evaluation and mitigation strategy (REMS) for clozapine (currently marketed as Clozaril, Versacloz, and generics), effective June 13, 2025. Clozapine, an antipsychotic medicine, can cause severe neutropenia (a low level of certain white blood cells), which can lead to serious and fatal infections. The removed REMS required enrollment of prescribers, pharmacies, and patients in a restricted distribution program and reporting of the level of certain white blood cells (i.e., the absolute neutrophil count (ANC)) to mitigate the risk of severe neutropenia.
https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/019758s107lbl.pdf?utm_medium=email&utm_source=govdelivery
Based on FDA’s re-evaluation of the Clozapine REMS and on the November 19, 2024 Joint Meeting of the Drug Safety and Risk Management Advisory Committee and the Psychopharmacologic Drugs Advisory Committee, the Agency determined that the REMS was no longer necessary to ensure the benefits of clozapine outweigh the risk of severe neutropenia. Although there remains a risk of severe neutropenia with clozapine use, clozapine labeling (including a new Medication Guide) is sufficient to mitigate this risk and maintain a positive benefit/risk profile. ANC monitoring can help identify neutropenia early to allow for timely intervention. Therefore, prescribers should continue to monitor patients’ ANC according to the monitoring frequencies described in the prescribing information. Eliminating the REMS is expected to improve access to clozapine and decrease the burden on the health care delivery system.
https://www.fda.gov/advisory-committees/advisory-committee-calendar/updated-meeting-time-and-public-participation-information-november-19-2024-joint-meeting-drug-safety?utm_medium=email&utm_source=govdelivery
CLINICAL PERSPECTIVES ++++ ++++ ++++
CLINICAL PERSPECTIVES
Impact of Approval of Pegcetacoplan for Rare Kidney Diseases
https://checkrare.com/impact-of-approval-of-pegcetacoplan-for-rare-kidney-diseases/
Carla Nester, MD, University of Iowa Stead Family Children’s Hospital and lead investigator of the VALIANT study, discusses the impact of the recent approval of pegcetacoplan (Empaveli) for patients with C3 glomerulopathy (C3G) and immune complex membranoproliferative glomerulonephritis (IC-MPGN).
Clinical Experience With Iptacopan for Treatment of PNH
https://checkrare.com/clinical-experience-with-iptacopan-for-treatment-of-pnh/
Jamie Koprivnikar, MD, Hematologist Oncologist at Hackensack University Medical Center, discusses her clinical experience with iptacopan for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).
Linvoseltamab in the Treatment of Relapsed/Refractory Multiple Myeloma
https://checkrare.com/linvoseltamab-in-the-treatment-of-relapsed-refractory-multiple-myeloma/
Joshua Richter, MD, Associate Professor of Medicine at the Tisch Cancer Institute, Director of Multiple Myeloma at the Blavatnik Family- Chelsea Medical Center at Mount Sinai, discusses the recent U.S. Food and Drug Administration (FDA) approval of linvoseltamab for the treatment of relapsed/refractory multiple myeloma (MM).
Evaluating Fenfluramine in Patients With Lennox-Gastaut Syndrome
https://checkrare.com/evaluating-fenfluramine-in-patients-with-lennox-gastaut-syndrome/
Amelie Lothe, PhD, Global Medical Community Head for Rare Epilepsies at UCB, discusses recent data evaluating fenfluramine in patients with Lennox-Gastaut syndrome.
Outpatient Administration of Naxitamab in Patients With Neuroblastoma
https://checkrare.com/outpatient-administration-of-naxitamab-in-patients-with-neuroblastoma/
Javier Oesterheld, MD, Division Chief of the Cancer and Blood Disorders Program at Levine Children’s Hospital and Founder and Executive Director of the ARISE Cancer Consortium, discusses outpatient administration of naxitamab in patients with neuroblastoma.
Immune Checkpoint Inhibition in Patients With Epstein-Barr Virus-Associated Gastric Cancer
https://checkrare.com/immune-checkpoint-inhibition-in-patients-with-epstein-barr-virus-associated-gastric-cancer/
James Hamrick, MD, Chairman of the Caris Precision Oncology Alliance, discusses outcomes of immune checkpoint inhibition in patients with Epstein-Barr virus-associated gastric cancer (EBVaGC).
Unmet Medical Needs in Rare Diseases
https://checkrare.com/unmet-medical-needs-in-rare-diseases/
Tobias Hagedorn, Secretary of the European Society for PKU (ESPKU), discusses unmet medical needs in rare disease communities.
Results From Amivanatamab Clinical Trial for Metastatic Colorectal Cancer
https://checkrare.com/results-from-amivanatamab-clinical-trial-for-metastatic-colorectal-cancer/
Dirk Arnold, MD, PhD, Director of the Asklepios Tumorzentrum Hamburg, discusses results from the OrigAMI-1 clinical trial in left-sided RAS/BRAF wild-type metastatic colorectal cancer (mCRC).
The ARISE Cancer Consortium
https://checkrare.com/the-arise-cancer-consortium/
Javier Oesterheld, MD, Division Chief of the Cancer and Blood Disorders Program at Levine Children’s Hospital and Founder and Executive Director of the ARISE Cancer Consortium, discusses the organization’s goals and upcoming clinical trials.
Risk Stratification in Patients With mIDH Glioma
https://checkrare.com/risk-stratification-in-patients-with-midh-glioma/
James Hamrick, MD, Chairman of the Caris Precision Oncology Alliance, discusses age in risk stratification in patients with mutant isocitrate dehydrogenase (mIDH) glioma.
Combination PD-1 Antibody and GPRC5D Antibody Therapy in Patients With Relapsed/Refractory Multiple Myeloma
https://checkrare.com/combination-pd-1-antibody-and-gprc5d-antibody-therapy-in-patients-with-relapsed-refractory-multiple-myeloma/
Paula Rodríguez-Ortero, MD, PhD, Hematologist at the University of Navarra, discusses results of PD-1 inhibitor combination therapy in patients with multiple myeloma (MM).
Pre-Diagnostic Symptoms and Time-to-Diagnosis in MLD
https://checkrare.com/pre-diagnostic-symptoms-and-time-to-diagnosis-in-mld/
Laura Adang, MD, PhD, Assistant Professor at Perelman School of Medicine at University of Pennsylvania, discusses a cohort study conducted analyzing pre-diagnostic symptoms and time-to-diagnosis in patients with metachromatic leukodystrophy (MLD).
CME PROGRAMS +++++++
CME PROGRAMS
Skeletal Involvement in Lysosomal Disorders
https://checkrare.com/learning/p-skeletal-involvement-in-lysosomal-disorders/
Ozlem Goker-Alpan, MD, Founder and President, Lysosomal & Rare Disorders Research & Treatment Center (LDRTC) and Ravi Kamath, MD, PhD, of Fairfax Radiological Consultants & Inova Health System and University of Virginia School of Medicine Fairfax, Virginia, USA discuss best practices to identify and treat bone problems associated with lysosomal disorders.
CREDITS: 1.0 CME | DURATION: 1.0 hours
EXPIRES: 6/30/2026 | FEE: $0
Fabry Disease Research Highlights
https://checkrare.com/learning/p-fabry-disease-research-highlights-2025/
Eric Wallace, MD, provides a summary of clinically relevant data presented at WORLDSymposium 2025 that can enhance the care of patients with Fabry disease.
CREDITS: 0.5 CME | DURATION: 0.5 hours
EXPIRES: 5/15/2026 | FEE: $0
Transforming Clinical Outcomes with Early Treatment of Lysosomal Disorders
https://checkrare.com/learning/p-transforming-clinical-outcomes-with-early-treatment-of-lysosomal-disorders/
Ozlem Goker-Alpan MD, David F. Kronn, MD, Uma Ramaswami FRCPCH, MD, Liz Jalazo, MD, and Lindsay Torrice MSN, CPNP-PC, examine the evidence available to address how to monitor, and possibly treat, children with lysosomal diseases that were diagnosed by newborn screening or soon after birth.
CREDITS: 1.00 CME | DURATION: 1 hour
EXPIRES: 4/03/2026 | FEE: $0
Consider Rare: Suspecting and Diagnosing Hereditary Angioedema
https://checkrare.com/learning/p-consider-rare-suspecting-and-diagnosing-hereditary-angioedema/
Jonathan A Bernstein, MD, describes the early symptoms of HAE and its clinical relevance, and discusses best practices to diagnose HAE more efficiently to reduce diagnostic delays.
CREDITS: 0.50 CME | DURATION: 30 minutes
EXPIRES: 3/15/2026 | FEE: $0
Hematologic Malignancies and Clinical Trial Participation: A Shared Decision-Making Approach
https://checkrare.com/learning/p-hematologic-malignancies-and-clinical-trial-participation-a-shared-decision-making-approach/
John Kuruvilla, MD, explores best practices for discussing possible clinical trial participation with patients who have hematologic malignancies.
CREDITS: 0.50 CME | DURATION: 30 minutes
EXPIRES: 11/30/2025 | FEE: $0
Complement-Mediated Kidney Disorders: A Case Series
https://checkrare.com/learning/p-complement-mediated-kidney-disorders-a-case-series/
Howard Trachtman, MD, FASN, and Carla M. Nester, MD, MSA, FASN, utilizes three unique case studies to address clinical questions which explore the complexities involved in diagnosing, treating, and managing patients with various complement-mediated kidney disorders, such as C3 glomerulopathy and atypical hemolytic uremic syndrome (aHUS).
CREDITS: 0.75 CME | DURATION: 45 minutes
EXPIRES: 11/15/2025 | FEE: $0
Cushing’s Syndrome Treatment Research Highlights: ENDO 2024
https://checkrare.com/learning/p-cushings-syndrome-treatment-research-highlights-endo-2024/
Maria Fleseriu, MD, FACE, Professor of Medicine and Neurological Surgery, Director of the Pituitary Center at Oregon Health & Science University, provides an overview of the latest clinical research presented at ENDO 2024 involving Cushing’s syndrome.
CREDITS: 0.5 CME | DURATION: 30 minutes
EXPIRES: 9/29/2025 | FEE: $0
FcRn and Myasthenia Gravis
https://checkrare.com/learning/p-fcrn-and-myasthenia-gravis/
Richard J. Nowak, MD, MS, explains the role of neonatal fragment crystallizable receptor (FcRn) in myasthenia gravis (MG) and how treatments that target FcRn are being used to manage patients with MG.
CREDITS: 0.5 CME | DURATION: 30 minutes
EXPIRES: 11/06/2025 | FEE: $0
RARE DISEASE LEARNING CENTERS +++++
RARE DISEASE LEARNING CENTERS
Neuroblastoma
https://checkrare.com/neuroblastoma/
Neuroblastoma is a rare childhood cancer, but it is the most common extracranial solid tumor in children. It is a neuroendocrine tumor that originates in neuroblasts or neural crest progenitor cells.
Rett Syndrome
https://checkrare.com/rett-syndrome/
Rett syndrome is a multisystem disorder that primarily affects girls. Only in rare cases are boys affected (who may experience more severe symptoms). Multiple loss-of-function mutations to the MECP2 gene are the cause of Rett syndrome.
Cutaneous T-Cell Lymphoma (CTCL)
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.
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.
Tenosynovial Giant Cell Tumor (TGCT)
https://checkrare.com/tenosynovial-giant-cell-tumor-tgct/
Tenosynovial giant cell tumor (TGCT) is a non-malignant tumor involving the joint synovium, bursae, and tendon sheath. These rare tumors are sometimes referred to as giant cell tumor of the tendon sheath (GCT-TS) and/or pigmented villonodular synovitis (PVNS).
Driving forwards in the management of OSA The Lancet Respiratory Medicine + +... +
https://www.thelancet.com/journals/lanres/article/PIIS2213-2600(25)00302-9/fulltext?dgcid=raven_jbs_etoc_email
Sep 2025
Volume 13Number 9p769-856, e45-e52
https://www.thelancet.com/journals/lanres/issue/vol13no9/PIIS2213-2600(25)X0009-6
Evaluation of the effect of multimorbidity on difficult-to-treat asthma using a novel score (MiDAS): a multinational study of asthma cohorts
https://www.thelancet.com/journals/lanres/article/PIIS2213-2600(25)00135-3/fulltext?dgcid=raven_jbs_etoc_feature_lanres
Turning the tide for rare disease
https://rarerevolutionmagazine.com/category/rare-insights/turning-the-tide-for-rare-disease/
Tobias Hagedorn, secretary of the European Society for Phenylketonuria (ESPKU)shares how they are challenging European legislation’s view of patient needs. By bridging policy, personal experience and scientific insight, they’re fighting to block proposed legislative changes that could redefine ‘unmet medical needs’ potentially impacting thousands and stalling research
https://rarerevolutionmagazine.com/leave-no-one-behind/
martes, 26 de agosto de 2025
Climate variability may speed up the spread of vector-borne diseases
https://www.news-medical.net/news/20250826/Climate-variability-may-speed-up-the-spread-of-vector-borne-diseases.aspx
Scientists warn that unpredictable climate swings could bring mosquito-borne disease outbreaks years earlier than expected, urging governments to act now on surveillance and preparedness.
Higher THC Concentrations Linked To Psychosis, Schizophrenia, Addiction © 2025 KFF. All rights reserved.
https://kffhealthnews.org/morning-breakout/higher-thc-concentrations-linked-to-psychosis-schizophrenia-addiction/
Higher THC Concentrations Linked To Psychosis, Schizophrenia, Addiction
Studies looking at therapeutic use linked to cancer showed mixed results in treating anxiety and depression, but over half the nontherapeutic studies showed links to unfavorable outcomes among healthy people. Plus: college students' mental health; mental health and cellphone use; and more.
MedPage Today: Potent Cannabis Products Linked To Psychosis, Mental Health Risks
Cannabis products containing high concentrations of delta-9-tetrahydrocannabinol (THC) -- the main psychoactive compound in marijuana -- were linked to psychosis, schizophrenia, and addiction, a systematic review found. Across 99 studies examining the effects of high-concentration THC products on mental health outcomes, 70% of the nontherapeutic studies (i.e., those not attempting to treat a medical condition or symptom) showed an unfavorable association with psychosis or schizophrenia and 75% found a relationship to cannabis use disorder (CUD). (Firth, 8/25)
In other mental health news —
Higher Ed Dive: Half Of College Students Say Their Mental Health Is ‘Fair’ To ‘Terrible,’ Survey Finds
Half of college students rate their mental health as fair, poor, or terrible, according to a recent survey from The Steve Fund, a nonprofit that focused on the mental health of young people of color. The survey also found about 40% of students were “very or extremely stressed about maintaining their mental health” while in college. About 1 in 5 students said the same about connecting with other students and finding their niche in college. (McLean, 8/26)
SciTechDaily: Owning A Smartphone Before 13 Linked To Alarming Mental Health Declines, Global Study Finds
A worldwide study involving more than 100,000 participants has found that receiving a smartphone before the age of 13 is linked with weaker mental health and lower overall wellbeing in early adulthood. The research, published in the peer-reviewed Journal of Human Development and Capabilities, reported that individuals aged 18 to 24 who first owned a smartphone at 12 or younger were more likely to experience suicidal thoughts, heightened aggression, feelings of detachment from reality, difficulties with emotional control, and diminished self-worth. (8/25)
KUTV: Audit Finds Utah Families Struggling With ‘Ghost Providers’ In Mental Health Care
A legislative audit has uncovered a troubling gap in Utah’s mental health care system, leaving families desperate for treatment for their children. The audit found that nearly 70% of providers listed in insurance directories are not actually accepting new patients. Lawmakers call them “ghost providers.” “They called up 180 providers in insurance directories and found out 69% of them were ghost providers, meaning they’re not accepting patients,” Rep. Steve Eliason, R-Sandy, said. “That’s a false promise.” (Harrison, 8/26)
AP: AI Inconsistent In Handling Suicide-Related Queries, Study Says
A study of how three popular artificial intelligence chatbots respond to queries about suicide found that they generally avoid answering questions that pose the highest risk to the user, such as for specific how-to guidance. But they are inconsistent in their replies to less extreme prompts that could still harm people. The study in the medical journal Psychiatric Services, published Tuesday by the American Psychiatric Association, found a need for “further refinement” in OpenAI’s ChatGPT, Google’s Gemini and Anthropic’s Claude. (Ortutay and O’Brien, 8/26)
The New York Times: Michael Phelps, Jay Glazer Bonded Over Mental Health. It Became A Star-Studded Support System
Though no one in the group is a mental health professional or has had training in the field, their personal experiences have allowed them to build a community. More than that, it’s tapped into a transformative idea: People often underestimate just how much they will enjoy deep conversations with other people. “Not because they fail to appreciate that having a meaningful conversation is something that they will enjoy personally,” said Nicholas Epley, a professor of behavioral science at the University of Chicago. “But because they underestimate how positively other people will respond to it. We underestimate how much power we actually have to make ourselves and other people feel better — notably better — by connecting with them.” (Devlin, 8/26)
On mental health care for first responders —
WABE: Marietta Police Trying Horse Therapy To Combat Officer Stress, Improve Mental Health And Policing
The Marietta Police Department recently started having officers take part in a horse therapy program to help their mental health and improve policing. (Mador, 8/24)
CBS News: Colorado Family Speaks Out About Mental Health Among First Responders After Northglenn Police Officer Dies By Suicide
Paul Gesi, a Northglenn police officer, died by suicide earlier this month after a long battle with PTSD. "Within probably the past year we started to see it weighing on him heavier as he would come home. and then it really got 'bad' really within the past two months," said Emily Gesi. "We saw signs of anxiety and depression. (We) never saw our father bring work home, but a little bit towards the end he started to talk about it a little bit more, about how he was starting to feel the darkness that he has been dealing with in his 41 years of being an officer." (Vidal, 8/25)
If you need help —
Dial 988 for 24/7 support from the Suicide & Crisis Lifeline. It's free and confidential.
lunes, 25 de agosto de 2025
The challenges of shift work on diet, sleep, and well-being
https://www.news-medical.net/news/20250825/The-challenges-of-shift-work-on-diet-sleep-and-well-being.aspx
From skipping meals to restless nights, new research exposes how shift schedules lead healthcare workers towards unhealthy habits, and what hospitals can do to help.
Where is childhood obesity growing fastest in England?
https://www.news-medical.net/news/20250825/Where-is-childhood-obesity-growing-fastest-in-England.aspx
Researchers reveal that whilst most of England faces worsening childhood obesity rates, a handful of London and South-East communities show slower growth, or even decline, highlighting the influence of deprivation, ethnicity, and local health policies in obesity trends.
Editorial Health insurance in southeast Asia: is it enough for UHC? The Lancet Regional Health – Southeast Asia + +... + +
The cognitive toll of household air pollution: cross–sectional associations between polluting cooking fuel use, cognitive functions and brain MRI in a rural aging population from Karnataka, India
https://www.thelancet.com/journals/lansea/article/PIIS2772-3682(25)00095-2/fulltext?dgcid=hubspot_update_feature_updatealerts_lansea&utm_campaign=update-lansea&utm_medium=email&_hsenc=p2ANqtz-8iYKoLp2C7YbsEczd5ZPckoQK3Dk2EXw9kLYrG7QyiljoneKQ8llwH78YCf0xdOSs5jOQJlTxJt8qLZuiEQCtRH0T12Q&_hsmi=377327971&utm_content=376922545&utm_source=hs_email
Editorial
Health insurance in southeast Asia: is it enough for UHC?
The Lancet Regional Health – Southeast Asia
https://www.thelancet.com/journals/lansea/issue/vol39nonull/PIIS2772-3682(25)X0007-X
Quantification of regional variation in ultra-processed food consumption and its sociodemographic correlates across Bangladesh, India, Pakistan, and Sri Lanka: insights from the South Asia Biobank
https://www.thelancet.com/journals/lansea/article/PIIS2772-3682(25)00104-0/fulltext?dgcid=hubspot_update_feature_updatealerts_lansea&utm_campaign=update-lansea&utm_medium=email&_hsenc=p2ANqtz-9RLlcpA4sRu9kY-fvT817fTKCocKHgMOji0Go6uCnaDrYko7nZ9bgiuGo1cpO5sXvIfXOQNt1LLIrp9njgaOXvUPmp8g&_hsmi=377327971&utm_content=376922545&utm_source=hs_email
Health insurance coverage among men and women in six countries within the Southeast Asia Region (2015–2022): a multilevel analysis of Demographic and Health Surveys
https://www.thelancet.com/journals/lansea/article/PIIS2772-3682(25)00105-2/fulltext?dgcid=hubspot_update_feature_updatealerts_lansea&utm_campaign=update-lansea&utm_medium=email&_hsenc=p2ANqtz-9wqcDXPDoAV68W3ZrMecBuERh15eLHjqdCNWapGqpi_-MkngIx1T6V55iVmCOMIk-lqcN9NtIX5P0YSLfxqQ_e4yQ6TA&_hsmi=377327971&utm_content=376922545&utm_source=hs_email
Cannabidiol and Liver Enzyme Level Elevations in Healthy Adults A Randomized Clinical Trial ++
https://jamanetwork.com/journals/jamainternalmedicine/fullarticle/2836267?utm_medium=email&utm_source=govdelivery
New CDER Research on the Effect of Cannabidiol on Liver Enzymes and Endocrine Measures Highlighted in CDER Impact Story
Despite limited and inconsistent human safety data, unregulated cannabidiol (CBD) products are widely available to consumers. In this CDER Impact Story, CDER experts describe a randomized, double-blinded, placebo-controlled study demonstrating that CBD use at doses representative of consumer products is associated with liver enzyme elevations in 5.6% of healthy adults. The study findings have important regulatory implications that can inform discussions about appropriate safeguards and underscore the need for further investigation of the long-term effects of CBD use, its effects on various populations, and the safety of lower doses commonly used by consumers.
CDER Investigators Address the Safety of CBD in a Randomized Trial
https://www.fda.gov/drugs/regulatory-science-action/cder-investigators-address-safety-cbd-randomized-trial?utm_medium=email&utm_source=govdelivery
domingo, 24 de agosto de 2025
Kennedy’s Anti-Vaccine Strategy Risks Forcing Shots Off Market, Manufacturers Warn By Stephanie Armour August 20, 2025
https://kffhealthnews.org/news/article/trump-rfk-maha-prasad-vaccine-injury-compensation-program-autism-allergies/?utm_campaign=KFF-This-Week&utm_medium=email&_hsenc=p2ANqtz-8gJBBKszuzKZ2tIffHgtIoLyZ6iU_UeoyvDZv-f0FurG9rROTkYTgd6PEnBqJckb7zsM1rGCqNr_4OCG97vWnLYIRYkw&_hsmi=377118209&utm_content=377118209&utm_source=hs_email
Kennedy’s Anti-Vaccine Strategy Risks Forcing Shots Off Market, Manufacturers Warn
Health and Human Services Secretary Robert F. Kennedy Jr. is targeting the government’s Vaccine Injury Compensation Program, without which manufacturers might cease producing shots.
How Will the 2025 Reconciliation Law Affect the Uninsured Rate in Each State? Allocating CBO’s Estimates of Additional Uninsured People Across the States Authors: Alice Burns, Jared Ortaliza, Justin Lo, Matthew Rae, and Cynthia Cox Published: Aug 20, 2025
https://www.kff.org/uninsured/how-will-the-2025-reconciliation-law-affect-the-uninsured-rate-in-each-state/
How Much the Uninsured Rate Could Rise in Each State
KFF's updated analysis apportions the increase in the number of uninsured across the 50 states and DC, including the percentage point increase by state. Nationally, CBO estimates that the reconciliation law will increase the number of uninsured by 10 million in 2034. That number would rise to over 14 million if enhanced premium tax credits for ACA Marketplace enrollees expire later this year.
The Problem Isn’t Trust in Vaccines, It’s That People Don’t Know Who to Trust Author: Drew Altman
https://www.kff.org/from-drew-altman/the-problem-isnt-trust-in-vaccines-its-that-people-dont-know-who-to-trust/?utm_campaign=KFF-This-Week&utm_medium=email&_hsenc=p2ANqtz--SiR7M4yU4akikokfSHaXKXaooOUMmNmeaBwbyNVO5N-3CcJRZY7QDewZ3eLpzYXC7fFwzmrq4sEOgMa8zi4Kixd-2sw&_hsmi=377118209&utm_content=377118209&utm_source=hs_email
In a new “Beyond the Data” column, KFF’s President and CEO Dr. Drew Altman analyzes years of KFF polling on vaccines in light of the current controversies about them. The real problem, he says, is not lack of public confidence in the safety of vaccines — few say they are unsafe — it’s that polarization and misinformation have eroded confidence in what’s true or not, and in scientific institutions people used to rely on for the facts.
Outcomes of Allogeneic HCT in Hodgkin Lymphoma in the Era of Checkpoint Inhibitors: A Joint CIBMTR and EBMT Analysis Authors: Miguel-Angel Perales, MD; Farrukh T. Awan, MD, MS; Ariane Boumendil, PhD; Jinalben Patel, BSc, MPH; Luca Castagna, MD; Emanuele Angelucci, MD; Herve Finel; Alexander D. Kulagin, MD; Bertram Glass, MD; Paolo Corradini, MD; Alex F. Herrera, MD; Didier Blaise, MD, HDR; Mohamed A. Kharfan-Dabaja, MD, MBA; Khalid Halahleh, MD; Sairah Ahmed, MD; Carmen Martínez, MD, PhD; Sebastian Giebel, MD, PhD; Silvia Montoto, MD, PhD; Richard J. Jones, MD; Nausheen Ahmed, MBBS; Ryan C. Lynch, MD; Marcos J. De Lima, MD; Mayzar Shadman, MD, MPH; Craig S. Sauter, MD; Kwang Woo Ahn, PhD; Mehdi Hamadani, MD; Ali Bazarbachi, MD, PhD; Anna Sureda, MD, PhD
sábado, 23 de agosto de 2025
viernes, 22 de agosto de 2025
Letting happiness happen eClinicalMedicine + +... +
https://www.thelancet.com/journals/eclinm/article/PIIS2589-5370(25)00400-6/fulltext?dgcid=raven_jbs_etoc_email
Aug 2025
Volume 86
https://www.thelancet.com/journals/eclinm/issue/vol86nonull/PIIS2589-5370(25)X0007-9
Global outbreaks of respiratory syncytial virus infections from 1960 to 2025: a systematic review and meta-analysis
https://www.thelancet.com/journals/eclinm/article/PIIS2589-5370(25)00284-6/fulltext?dgcid=raven_jbs_etoc_feature_eclinm
Diagnostic and phylogenetic perspectives of the 2023 Murray Valley encephalitis virus outbreak in Australia: an observational study + +... +
https://www.thelancet.com/journals/lanmic/article/PIIS2666-5247(25)00017-5/fulltext?dgcid=hubspot_update_feature_updatealerts_lanmic&utm_campaign=update-lanmic&utm_medium=email&_hsenc=p2ANqtz-8x7hQijBp45brh-Iwjg5GdGdXny6dXwbED-_7NYR7MlVaf_jwRI6y2-QsJ8tEnPFNusZ_zgdGFyTyEoHXYqDEkzofZug&_hsmi=376629003&utm_content=376340242&utm_source=hs_email
Editorial
SARS-CoV-2 origins: imperfect but clear evidence
The Lancet Microbe
https://www.thelancet.com/journals/lanmic/issue/vol6no8/PIIS2666-5247(25)X0008-2
Efficacy, safety, pharmacokinetics, and associated microbiome changes of ibezapolstat compared with vancomycin in adults with Clostridioides difficile infection: a phase 2b, randomised, double-blind, active-controlled, multicentre study
https://www.thelancet.com/journals/lanmic/article/PIIS2666-5247(25)00054-0/fulltext?dgcid=hubspot_update_feature_updatealerts_lanmic&utm_campaign=update-lanmic&utm_medium=email&_hsenc=p2ANqtz--1Yh_0NN8mMDW14zVN9nZjEC9tK6JXdtL69Liry_4-vuWqI7tF5vxmW7Sd9vNcJppCBnf9VymAMIl7AMM_hlg1dTYOkQ&_hsmi=376629003&utm_content=376340242&utm_source=hs_email
New Technique for Measuring DNA Damage Could Improve Cancer Therapy and Radiological Emergency Response August 21, 2025
https://www.nist.gov/news-events/news/2025/08/new-technique-measuring-dna-damage-could-improve-cancer-therapy-and
New NIST Method Precisely Measures Radioactivity in Tiny Samples
https://www.nist.gov/news-events/news/2025/07/new-nist-method-precisely-measures-radioactivity-tiny-samples
Vaccines can help keep you healthy Ask your VA provider about your vaccine options. ++++...
https://news.va.gov/141992/vaccines-can-help-keep-you-healthy/
Veteran says VA Health Connect saved his life
‘Call as soon as you have a doubt!’
https://news.va.gov/141962/veteran-says-va-health-connect-saved-his-life/
How to navigate VA’s new secure sign-in changes
VA.gov sign-in changes
https://news.va.gov/141782/how-to-navigate-vas-new-secure-sign-in-changes/
VA breaks ground on its first PACT Act construction initiative in Rolla, MO
https://news.va.gov/news/health/
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