Clinical Insights
Generalized Myasthenia Gravis: AANEM 2026 Highlights
The American Association of Neuromuscular and Electrodiagnostic Medicine (AANEM) held its 2026 annual meeting September 29–October 1 in Orlando. CheckRare focused its meeting coverage on generalized myathesthenia gravis (gMG), and its treatment and management.Data on...
Miv-cel Shows Durable Results in Stiff Person Syndrome and Myasthenia Gravis
Naji Gehchan, MD, MBA, Chief Medical and Development Officer at Kyverna Therapeutics, discusses one-year data showing sustained benefits of KYV-101 (mivocabtagene autoleucel ; Miv-cel) in stiff person syndrome (SPS), along with longer-term follow-up data in generalized myasthenia gravis (gMG).
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.
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.
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Uplizna (inebilizumab): A First-In-Class Approach to Generalized Myasthenia Gravis Treatment
Richard Nowak, MD, Director of the Myasthenia Gravis Clinic at Yale University, discusses the recent U.S. Food and Drug Administration (FDA) approval of Uplizna (inebilizumab) for the treatment of...
FDA Approved Darzalex Faspro Combination Therapy for Patients With Transplant Ineligible Newly Diagnosed Multiple Myeloma
The US Food and Drug Administration (FDA) has approved Darzalex Faspro (daratumumab and hyaluronidase) in combination with bortezomib, lenalidomide, and dexamethasone (D-VRd) for the treatment of...
Bleximenib Combination Therapy for Patients With Acute Myeloid Leukemia
Hartmut Döhner, MD, Professor of Medicine and Medical Director of the Department of Hematology and Oncology, Ulm University, Germany, discusses bleximenib combination therapy for treatment of...
New Formulation of Berotralstat for Treatment of Pediatric Patients With Hereditary Angioedema
Raffi Tachdjian, MD, Associate Clinical Professor of Medicine & Pediatrics, Division of Allergy & Clinical Immunology, University of California Los Angeles, discusses a new formulation of...
FDA Expands Indication for Cerezyme (Imiglucerase) To Treat Gaucher Disease Type 3
The US Food and Drug Administration (FDA) has approved the expanded indication of Cerezyme (imiglucerase) for the treatment of non-neuronal symptoms in adult and pediatric patients with Gaucher...
Rare Diseases in Ireland – New Efforts to Improve Access to Care
Each country takes a different approach to rare diseases, from the way it defines the term to the health policies it implements to its approach to research. In Ireland, as in the rest of Europe,...
Data From the CORAL Trial of Nalbuphine ER in Patients With Idiopathic Pulmonary Fibrosis
Data from the Phase 2b CORAL trial of nalbuphine extended release (ER) for the treatment of chronic cough in patients with idiopathic pulmonary fibrosis (IPF) was recently published in the Journal...
SWITCH Clinical Trial Design for Patients With Hemophilia A
Guy Young, MD, Children’s Hospital Los Angeles, discusses the SWITCH clinical trial design for patients with hemophilia A. Hemophilia A is an inherited bleeding disorder in which the blood...
How the TED Community Organization Helps Patients With Thyroid Eye Disease Take Their Lives Back
Christine Gustafson, Executive Director and CEO of the TED Community Organization, discusses her personal journey with thyroid eye disease (TED) and why she started the TED Community Organization. ...
Phase 2 Results of Dusquetide in the Treatment of Oral Ulcers Caused by Behçet’s Disease
Recently, results from a phase 2a study evaluating SGX945 (dusquetide) for the treatment of Behçet’s disease were published in Rheumatology (Oxford). Behçet’s disease is a rare vasculitis...
Week 52 Results of TransCon CNP (Navepegritide) in Children With Achondroplasia
Janet Legare, MD, Professor of Pediatrics in the Division of Genetics at the University of Wisconsin, discusses week 52 results of TransCon CNP (navepegritide) in children with achondroplasia....
Real World Safety Data of Enjaymo (Sutimlimab) in Patients With Cold Agglutinin Disease
Catherine M. Broome, MD, Professor of Medicine and Director of Cellular Apheresis, Lombardi Cancer Center, MedStar Georgetown University, discusses real world safety data of Enjaymo (sutimlimab) in...
Long-term Efficacy of Rilzabrutinib in Patients With Immune Thrombocytopenia
David Kuter, MD, DPhil, Professor of Medicine at Harvard Medical School, Massachusetts General Hospital, discusses treatment with rilzabrutinib in patients with immune thrombocytopenia (ITP). ...
Recent Data on Ravulizumab in Rare Hematologic Conditions
Anita Hill, MD, PhD, Global Medical Head for Hematology & Nephrology and Transplant at Alexion, AstraZeneca Rare Disease, discusses recent data on ravulizumab in rare hematologic conditions....
Open-Label Extension Study Testing Arimoclomol in Patients With NPC
A recent paper published in Molecular Genetics and Metabolism presented safety and efficacy outcomes from the 48-month open-label extension of the NPC-002 study of arimoclomol in patients with...
FDA Grants Fast Track Designation to Myasthenia Gravis Therapy
Jan Klatt, MD, Head of Development Unit Neurology & Immunology, Merck Healthcare, Germany, discusses the U.S. Food and Drug Administration’s (FDA) Fast Track designation of cladribine capsules...
FDA Approves First Treatment for Menkes Disease
The U.S Food and Drug Administration (FDA) has approved Zycubo (copper histidinate) for the treatment of pediatric patients with Menkes disease. Menkes disease is a rare X-linked pediatric disease...
A Family’s Experience With Nasopharyngeal Carcinoma
Floyd Stewart, patient with stage 4 nasopharyngeal carcinoma, and Monique Stewart, advocate and Floyd’s wife, discuss their family’s experience with the disease and their advocacy work. ...
Post Hoc Analysis of Long-Term Efficacy of Migalastat in Females With Fabry Disease
A recent post hoc analysis of long-term efficacy of migalastat in females with Fabry disease was published in the Journal of Medical Genetics. Fabry disease is a rare lysosomal storage disease...
FDA Expands Cablivi (Caplacizumab) Indication to Patients Ages 12 Years and Older With aTTP
The U.S. Food and Drug Administration (FDA) has approved Cablivi (caplacizumab) for the treatment of pediatric patients ages 12 years and older with acquired thrombotic thrombocytopenic purpura...
Reproduction and Pregnancy in Lysosomal Disorders
Best practices to manage pregnancy and other reproductive issues associated with lysosomal disorders (LDs)
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TETON-2 Findings in Treatment of Idiopathic Pulmonary Fibrosis #podcast #raredisease
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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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