Clinical Insights
Gambling on Clinical Trials | The CheckRare Brief
In this episode, we discuss gambling on clinical trials, the FDA approval of a new formulation of Sjögren’s disease treatment, and an approval in IgA nephropathy. FDA Approves a New Liquid Formulation for Sjögren's Disease The FDA has approved a new...
Clinical Trials for Rare Endocrine Disorders | Rare Endocrine Exchange
In this episode, endocrinologist Margarita Ochoa-Maya, MD, speaks with NIH endocrinologist Katherine Araque, MD, about clinical trials for rare endocrine disorders. The conversation examines clinical trials from three perspectives: patients, clinical researchers, and sponsors.
FDA Grants Accelerated Approval to Ravulizumab for Adults With Primary Immunoglobulin A Nephropathy
The US Food and Drug Administration (FDA) has granted accelerated approval to Ultomiris (ravulizumab) for the treatment of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN).
Z-Basivarsen Shows Functional Improvements in One-Year Data for Myotonic Dystrophy Type 1
Laurie Gutmann, MD, professor of neurology at Indiana University, discusses data showing functional improvements with zeleciment basivarsen (z-basivarsen; DYNE-101) in patients with myotonic dystrophy type 1.
More
FDA Approves Selumetinib to Treat Patients With Neurofibromatosis Type 1
The U.S. Food and Drug Administration (FDA) has approved Koselugo (selumetinib) for the treatment of adult patients with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform...
Updates to Prescribing Information for Elevidys in Treatment of Duchenne Muscular Dystrophy
The U.S. Food and Drug Administration (FDA) has updated the prescribing information for Elevidys (delandistrogene moxeparvovec) for Duchenne muscular dystrophy (DMD). DMD is a rare genetic,...
Approval of Treatment for Patients With Familial Chylomicronemia Syndrome (FCS)
The U.S. Food and Drug Administration (FDA) has approved Redemplo (plozasiran) for the treatment of adult patients with familial chylomicronemia syndrome (FCS). FCS is an inherited condition that...
FDA Approves Ziftomenib for Relapsed/Refractory NPM1-Mutated Acute Myeloid Leukemia
The U.S. Food and Drug Administration (FDA) has approved Komzifti (ziftomenib) for the treatment of relapsed or refractory (R/R) acute myeloid leukemia (AML) with susceptible NPM1 mutation. Acute...
Ongoing Phase 3 Study of Paltusotine in Carcinoid Syndrome Due to Neuroendocrine Tumors
Aman Chauhan, MD, Oncologist at the University of Miami, discusses details of a phase 3 study testing paltusotine in patients with carcinoid syndrome due to neuroendocrine tumors (NETs). ...
The Shift to Precision Medicine in Myasthenia Gravis
Alexandra C. Bayer Wildberger, PhD, Post-Doctoral Associate at Yale School of Medicine, discusses the shift to precision medicine in myasthenia gravis (MG). MG is a chronic autoimmune...
Updates in IPF Chronic Cough From CHEST 2025
Philip Molyneaux, MD, PhD, a Professor of Interstitial Lung Disease (ILD) at Imperial College London and the Asthma+Lung UK Chair of Respiratory Research at Royal Brompton Hospital, where is also...
Long-Term Data From the AQUARIUS Study of Avacopan in Patients With GPA and MPA
Naomi Patel, MD, MPH, Rheumatologist at Massachusetts General Hospital, discusses long-term data from the AQUARIUS study of avacopan in patients with granulomatosis with polyangiitis (GPA) and...
Data on the RAISE Clinical Trial Program of Zilucoplan for the Treatment of Myasthenia Gravis
Michael Weiss, MD, Neurologist at the University of Washington, discusses data on the RAISE and RAISE-XT clinical trials of zilucoplan for the treatment of generalized myasthenia gravis (gMG)....
New Data on Vyvgart for Patients With AChR-Ab Seronegative Myasthenia Gravis
James Howard, Jr., MD, Professor of Neurology at the University of North Carolina, discusses new data on the safety and efficacy of Vyvgart (efgartigimod alfa) for patients with AChR-Ab seronegative...
FDA Approves Darzalex Faspro for Patients With High-Risk Smoldering Multiple Myeloma
The U.S. Food and Drug Administration (FDA) has approved Darzalex Faspro (daratumumab and hyaluronidase-fihj) for the treatment of adult patients with high-risk smoldering multiple myeloma (HR-SMM)....
FDA Approves First Treatment for Thymidine Kinase 2 Deficiency
The U.S. Food and Drug Administration (FDA) has approved Kygevvi for the treatment of adults and pediatric patients, with an age of symptom onset on or before 12 years, with thymidine kinase 2...
A Patient’s Diagnostic Journey With Systemic Mastocytosis
Joan Smith, a patient with systemic mastocytosis, discusses her diagnostic journey with systemic mastocytosis (SM). SM is a rare disease, usually caused by mutations in the KIT D816V gene....
Results From the DeFi Clinical Trial of Nirogacestat in Patients With Desmoid Tumors
Bernd Kasper, MD, PhD, University of Heidelberg Cancer Center, discusses results from the DeFi clinical trial of nirogacestat in patients with desmoid tumors. Desmoid tumors are...
Results from the ElevAATe Clinical Trial of Efdoralprin Alfa for Patients With AAT Deficiency
Alaa Hamed, MD, Global Head of Medical Affairs Rare Diseases at Sanofi, discusses results from the ElevAATe clinical trial of efdoralprin alfa for the treatment of patients with alpha-1 antitrypsin...
Long-Term Analysis of Pimicotinib for the Treatment of Patients With TGCT
Xiaohui Niu, Director of the Bone and Soft Tissue Tumour Diagnosis and Research Centre at Beijing Jishuitan Hospital, discusses a long-term analysis of the MANEUVER clinical trial of pimicotinib for...
FDA Expands Indication of Sotatercept for Patients With Pulmonary Arterial Hypertension
The U.S. Food and Drug Administration (FDA) has approved an update to the product label of Winrevair (sotatercept) for the treatment of adults with pulmonary arterial hypertension (PAH). PAH is a...
Results from the TEASE-2 Clinical Trial of Gildeuretinol in Patients With Stargardt Disease
Philip J. Ferrone, MD, Vitreoretinal Consultants of New York, discusses results from the TEASE-2 clinical trial of gildeuretinol in patients with Stargardt disease. Stargardt disease...
FDA Approves Revumenib for the Treatment of Relapsed/Refractory Acute Myeloid Leukemia
The U.S. Food and Drug Administration (FDA) has approved Revuforj (revumenib) for the treatment of relapsed/refractory acute myeloid leukemia (AML). The indication is for adult and pediatric...
The Genetics of Epilepsy: The Importance of Identifying Underlying Causes
Accurate diagnosis and treatment of epilepsy challenges the medical community, patients, and their loved ones.[1] As many as one third of patients have seizures that are inadequately controlled with...
Systemic Mastocytosis: Recognition, Diagnosis, and Clinical Management
Highlighting the early signs of Systemic Mastocytosis (SM), criteria and tools, and the importance of timely referral/testing
Recent Videos
TETON-2 Findings in Treatment of Idiopathic Pulmonary Fibrosis #podcast #raredisease
Inhaled Treprostinil Reduces Multiple Disease Progression Events in IPF
Highlights from The Liver Meeting 2026 | The CheckRare Brief
Results of the Phase 3 OASIS Study of Oral Olorofim
Social Wall
What should you know about myasthenia gravis?
From understanding the underlying disease mechanisms to exploring FDA-approved treatment options and ongoing clinical research, staying informed is essential for the rare disease community.
The Myasthenia Gravis Disease Learning CenterRead more brings together educational information and resources to help patients, caregivers, and healthcare professionals better understand this complex condition.
Explore the learning center: https://checkrare.com/myasthenia-gravis/
#MyastheniaGravis #RareDiseaseEducation #HealthcareProfessionals #ClinicalResearch #PatientEducation
UCB Study Highlights the Burden of Ocular Myasthenia Gravis
Living With Ocular Symptoms of Myasthenia Gravis
Steven Nathan, MD, MBBCh, FCCP, Director of the Advanced Lung Disease and Lung Transplant Program at Inova Fairfax Hospital, discusses findings from a post-hoc analysis of the phase 3 TETON-2 study, presented at the CHEST 2026 Annual Meeting.
View the full interview at Read morehttps://checkrare.com/inhaled-treprostinil-reduces-multiple-disease-progression-events-in-ipf/
What's the latest research in rare liver conditions?
Get updates from The Liver Meeting 2026 and more on this week's episode of The CheckRare Brief at https://checkrare.com/highlights-from-the-liver-meeting-2026-the-checkrare-brief/
What's the latest research in rare liver conditions?
Get updates from The Liver Meeting 2026 and more on this week's episode of The CheckRare Brief at https://checkrare.com/highlights-from-the-liver-meeting-2026-the-checkrare-brief/
TETON-2 Findings in Treatment of Idiopathic Pulmonary Fibrosis #podcast #raredisease
Inhaled Treprostinil Reduces Multiple Disease Progression Events in IPF
Highlights from The Liver Meeting 2026
Inhaled Treprostinil Reduces Multiple Disease Progression Events in IPF
Highlights from The Liver Meeting 2026 | The CheckRare Brief
Johan Maertens, MD, PhD, Professor of Hematology at University Hospitals Leuven (UZ Leuven) in Belgium and Principal Investigator of the OASIS study, discusses positive phase 3 trial results evaluating oral olorofim in patients with invasive aspergillosis.
View the full interview at Read morehttps://checkrare.com/phase-3-oasis-study-shows-promise-of-oral-olorofim-for-invasive-aspergillosis/
Johan Maertens, MD, PhD, Professor of Hematology at University Hospitals Leuven (UZ Leuven) in Belgium and Principal Investigator of the OASIS study, discusses positive phase 3 trial results evaluating oral olorofim in patients with invasive aspergillosis.
View the full
Phase 3 OASIS Study Shows Promise of Oral Olorofim for Invasive Aspergillosis
Results of the Phase 3 OASIS Study of Oral Olorofim
UCB Study Highlights the Burden of Ocular Myasthenia Gravis
CheckRare 4 hours ago