Clinical Insights
FDA Approves Enspryng, the First At-Home Treatment Option for Thyroid Eye Disease
The US Food and Drug Administration (FDA) has approved Enspryng (satralizumab) for the treatment of adults with thyroid eye disease (TED). TED is a chronic endocrine, autoimmune disease characterized by immune-mediated orbital inflammation that greatly...
Ultragenyx Gets $203M in Second Priority Review Voucher This Month | The CheckRare Brief
On this episode, we discuss the FDA approval of Enspryng for TED, the new FDA Rare Disease Framework Initiative, Ultrangenyx’s second sold PRV this month, and using AI to reduce diagnostic delays.
Efficacy of Vosoritide to Treat Hypochondroplasia
Andrew Dauber, MD, MMsc, Chief of Endocrinology at Children’s National, discusses the efficacy of vosoritide to treat persons with hypochondroplasia.
Efficacy of Infigratinib on Achondroplasia
Daniela Rogoff, MD, PhD, InChief Medical Officer, Skeletal Dysplasia of BridgeBio, discusses the efficacy of infigratinib to treat persons with achondroplasia.
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New Data on Pegcetacoplan in Patients With C3G and IC-MPGN
Fadi Fakhouri, MD, PhD, Professor of Nephrology at CHUV Lausanne, Switzerland, discusses new data on pegcetacoplan in patients with C3 glomerulopathy (C3G) and immune complex-mediated...
FDA Approves Linvoseltamab for Treatment of Multiple Myeloma
The U.S. Food and Drug Administration (FDA) has granted accelerated approval to Lynozyfic (linvoseltamab-gcpt) for the treatment of adults with relapsed or refractory multiple myeloma (MM) who have...
Delpacibart Braxlosiran in Patients With Facioscapulohumeral Muscular Dystrophy
Mike Flanagan, PhD, Chief Scientific Officer at Avidity Biosciences, discusses topline results from study testing delpacibart braxlosiran (del-brax) in patients with facioscapulohumeral muscular...
FDA Approves Therapy for Primary Immunodeficiencies
The U.S. Food and Drug Administration (FDA) has approved Gammagard Liquid ERC (immune globulin infusion) for patients ages two years and older with primary immunodeficiencies. Primary...
Ibrutinib Plus Venetoclax in Patients With Chronic Lymphocytic Leukemia and Small Lymphocytic Lymphoma
Paolo Ghia, MD, PhD, Professor of Medical Oncology at Università Vita-Salute San Raffaele in Milan, Italy, discusses results from clinical trial testing ibrutinib plus venetoclax in patients with...
Eosinophilic Esophagitis Explained
Marc Rothenberg, MD, of the Consortium of Eosinophilic Gastrointestinal Disease Researchers (CEGIR), and Professor of Medicine at Cincinnati Children’s Hospital, gives an overview of eosinophilic...
The Genetic Disorders Mucociliary Clearance Consortium
Stephanie Davis, MD, and Thomas Ferkol, MD, Professors of Medicine at University of North Carolina at Chapel Hill, discuss the value of being part of the Rare Diseases Clinical Research Network...
FDA Approves Emapalumab to Treat Still’s Disease
The U.S. Food and Drug Administration (FDA) has approved Gamifant (emapalumab-lzsg) for the treatment of patients with hemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS)...
First Results From a Study Evaluating CAR-T Cell Therapy for Large B Cell Lymphoma
Krish Patel, MD, Medical Oncologist at Sarah Cannon Research Institute, discusses first results from a study evaluating CAR-T cell therapy for large B cell lymphoma (LBCL). LBCL is a...
Results from the VERIFY Clinical Trial in Polycythemia Vera
Andrew T. Kuykendall, MD, Associate Member in the Department of Hematology at Moffitt Cancer Center, discusses results from the VERIFY clinical trial in polycythemia vera (PV). PV is a...
Results From the CEPHEUS Clinical Trial in Multiple Myeloma
Saad Usmani, MD, Myeloma Specialist and Cellular Therapist at Memorial Sloan Kettering Cancer Center, discusses results from the CEPHEUS clinical trial in multiple myeloma (MM). MM is...
Efficacy of Amivanatamab to Treat Left-Sided RAS/BRAF Wild-Type Metastatic Colorectal Cancer
Dirk Arnold, MD, PhD, Director of the Asklepios Tumorzentrum Hamburg, discusses the OrigAMI-2 clinical trial in left-sided RAS/BRAF wild-type metastatic colorectal cancer (mCRC). ...
Results from the CARTITUDE-1 Study in Patients With Multiple Myeloma
Peter Voorhees, MD, Atrium Health and Levine Cancer Institute, Wake Forest University School of Medicine, discusses results from the CARTITUDE-1 study in patients with multiple myeloma (MM). ...
Cutaneous T-Cell Lymphoma: Overview, Management, and Quality-of-Life
Neha Mehta-Shah, MD, Medical Oncologist at Washington University St. Louis, provides an overview of cutaneous T-cell lymphoma (CTCL), discusses management options, and addresses quality-of-life...
FDA Approves Nitisinone for the Treatment of Alkaptonuria
The U.S. Food and Drug Administration (FDA) has approved Harliku (nitisinone) for the treatment of adult patients with alkaptonuria. Alkaptonuria (AKU) is an ultra-rare inherited condition that...
Implications of Nipocalimab Approval on Pediatric Patients With Myasthenia Gravis
Jonathan Strober, MD, Director of the Neuromuscular Clinic and Professor of Pediatrics and Neurology at the University of California at San Francisco, discusses the implications of nipocalimab's...
FDA Approves Dupixent for the Treatment of Bullous Pemphigoid
The U.S. Food and Drug Administration (FDA) has approved Dupixent (dupilumab) for the treatment of adult patients with bullous pemphigoid. Bullous pemphigoid is a rare autoimmune, skin disorder...
New Data From EMBARK Study in Patients With Duchenne Muscular Dystrophy Receiving Gene Therapy
Aravindhan Veerapandiyan, MD, Pediatric Neuromuscular Neurologist at Arkansas Children’s Hospital, discusses new data from the EMBARK part 2 study of Elevidys (delandistrogene moxeparvovec) in...
Phase 3 GLISTEN Trial in Patients With Primary Biliary Cholangitis
Gideon Hirschfield, MD, of the Toronto General Hospital and Toronto Centre for Liver Disease, discusses results from the phase 3 GLISTEN trial in patients with primary biliary cholangitis (PBC)....
Chronic Myeloid Leukemia: Overview, Treatment Options, and Advice
Jorge Cortes, MD, Director of the Georgia Cancer Center, provides an overview of chronic myeloid leukemia (CML), discusses treatment options, and offers advice to newly diagnosed patients. ...
Neuroblastoma
Neuroblastoma is a rare childhood cancer characterized by a neuroendocrine tumor originating in neuroblasts or neural crest progenitor cells.
Recent Videos
TETON-2 Findings in Treatment of Idiopathic Pulmonary Fibrosis #podcast #raredisease
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Results of the Phase 3 OASIS Study of Oral Olorofim
Phase 3 OASIS Study Shows Promise of Oral Olorofim for Invasive Aspergillosis
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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
Phase 3 OASIS Study Shows Promise of Oral Olorofim for Invasive Aspergillosis
🧬 A new chapter in gene therapy for spinal muscular atrophy (SMA).
How is Itvisma changing the treatment landscape for people living with SMA? In this CheckRare feature, John Day, MD, PhD, of Stanford University, discusses the development of this one-time gene therapy, its targeted Read moredelivery approach, and clinical trial findings on motor function.
Discover what this advancement could mean for the future of SMA treatment.
🔗 Read the full article: https://checkrare.com/itvisma-gene-therapy-for-patients-with-spinal-muscular-atrophy/
#CheckRare #SpinalMuscularAtrophy #SMA #RareDisease #GeneTherapy #MedicalInnovation
🧬 A new chapter in gene therapy for spinal muscular atrophy (SMA).
How is Itvisma changing the treatment landscape for people living with SMA? In this CheckRare feature, John Day, MD, PhD, of Stanford University, discusses the development of this one-time gene therapy, its
Living With Ocular Symptoms of Myasthenia Gravis
CheckRare 49 minutes ago