Clinical Insights
How Bispecific Antibodies Are Changing Multiple Myeloma Treatment
Nisha Joseph, MD, Associate Professor in the Department of Hematology and Medical Oncology at the Emory University School of Medicine, discusses how bispecific antibodies are changing multiple myeloma treatment. What Is Multiple Myeloma?...
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.
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).
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FDA Approves Oral Prophylactic Therapy (berotralstat) for Pediatric Patients With Hereditary Angioedema
The U.S. Food and Drug Administration (FDA) has approved an oral pellet formulation of Orladeyo (berotralstat) for prophylactic therapy in pediatric patients with hereditary angioedema (HAE) ages 2...
Long-Term Data With Dawnzera for the Treatment of Hereditary Angioedema
Michael Manning, MD, Allergist and Immunologist, discusses long-term data with Dawnzera (donidalorsen) for the treatment of patients with hereditary angioedema (HAE). HAE is a...
Catching the Clues, Changing the Course of Lysosomal Storage Disorders
Catching the Clues, Changing the Course of Lysosomal Storage Disorders Professor Yoshikatsu Eto, Dr. Nicole Muschol, Professor Patrício Aguiar, Dr. Robert HopkinThis interactive symposium explores...
Effects of Vutrisiran on Cardiac Structure and Function in Patients With Transthyretin Amyloidosis With Cardiomyopathy
A recent study, published in Nature Medicine, looked at the effects of vutrisiran on cardiac structure and function in patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM) through a...
FDA Approves Gene Therapy (Waskyra) for Patients with Wiskott-Aldrich Syndrome
The U.S. Food and Drug Administration (FDA) has approved Waskyra (etuvetidigene autotemcel) for the treatment of patients ages 6 months and older with Wiskott-Aldrich syndrome (WAS). WAS is a...
Advancing Thyroid Eye Disease Care: Integrating Tepezza, New Technology, and Optimizing Multidisciplinary Teams
Maja Kostic, MD, PhD, neuro-ophthalmologist at Bascom Palmer Eye Institute and Assistant Professor of Ophthalmology at the University of North Carolina at Chapel Hill, discusses advancing thyroid...
FDA Adds Marginal Zone Lymphoma to Indication of Breyanzi
The U.S Food and Drug Administration (FDA) has approved Breyanzi (lisocabtagene maraleucel; liso-cel) for the treatment of adults with relapsed/refractory marginal zone lymphoma (MZL) who have...
One-Year Phase 3 Data Testing Empaveli (pegcetacoplan) in Patients with C3G and IC-MPGN
Carla Nester, MD, Professor of Pediatrics-Nephrology at the University of Iowa, discusses one-year phase 3 data of Empaveli (pegcetacoplan) for treatment of C3 glomerulopathy (C3G) and immune...
Interim Results From Study of Mezagitamab for the Treatment of IgA Nephropathy
Jonathan Barratt, Mayer Professor of Renal Medicine at the University of Leicester, discusses interim results from a study testing mezagitamab for the treatment of immunoglobulin A (IgA)...
Kygevvi for Patients With Thymidine Kinase 2 Deficiency
Kim Moran, Head of U.S. Rare Diseases at UCB, discusses the approval of Kygevvi (doxecitine and doxribtimine) for patients with thymidine kinase 2 deficiency (TK2d). TK2d is a rare...
Data from the DUPLEX Study of Sparsentan in Focal Segmental Glomerulosclerosis
Michelle Rheault, MD, Pediatric Nephrologist at the University of Minnesota, discusses data from the DUPLEX study testing sparsentan to treat patients with focal segmental glomerulosclerosis (FSGS)....
FDA Approves Voyxact for Primary Immunoglobulin A Nephropathy
The U.S. Food and Drug Administration (FDA) has approved Voyxact (sibeprenlimab) for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk for disease...
FDA Approves Intrathecal Gene Therapy for Patients with Spinal Muscular Atrophy
The U.S. Food and Drug Administration (FDA) has approved Itvisma (onasemnogene abeparvovec) for the treatment of patients ages two years and older with spinal muscular atrophy (SMA) with a confirmed...
The Development of Nipocalimab for the Treatment of Sjögren’s Disease
Ghaith Noaiseh, MD, Associate Professor of Allergy, Clinical Immunology, and Rheumatology at The University of Kansas Medical Center, discusses the development of nipocalimab for the treatment of...
ORIGIN 3 Clinical Trial Evaluating Atacicept in Adults With IgA Nephropathy
Richard Lafayette, MD, Professor of Medicine at Stanford University, discusses the ORIGIN clinical trial evaluating atacicept in adults with IgA nephropathy (IgAN). IgAN is a kidney...
FDA Approves Hyrnua for HER2-Positive Non-Small Cell Lung Cancer
The U.S. Food and Drug Administration (FDA) has approved Hyrnua (sevabertinib) for the treatment of adults with locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) with...
The Use of Nipocalimab in Adolescent Patients With Generalized Myasthenia Gravis
Jonathan Strober, MD, pediatric neurologist and neuromuscular specialist at the University of California at San Francisco, discusses the use of nipocalimab in adolescent patients with generalized...
Positive Topline Results from the CALIBRATE Trial of Encaleret in Patients With Autosomal Dominant Hypocalcemia Type 1
Michael A. Levine, MD, ML, Children’s Hospital of Philadelphia, discusses positive topline results from the CALIBRATE trial of encaleret in patients with autosomal dominant hypocalcemia type 1...
EPIC Clinical Trial Design: Evaluating Nipocalimab Versus Efgartigimod in Patients With Myasthenia Gravis
Nolan Campbell, PhD, U.S. Medical Director at Johnson & Johnson, discusses the EPIC clinical trial design that will evaluate the efficacy of nipocalimab versus efgartigimod in patients with...
Efficacy of Nipocalimab in Generalized and Ocular gMG: Long-Term Outcomes and Phase 3 VIVACITY-MG3 Findings
Sindhu Ramchandren, MD, Executive Medical Director of Neuroscience at Johnson & Johnson, discusses long-term efficacy of nipocalimab in patients with generalized myasthenia gravis (gMG). ...
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
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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/
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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
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