Clinical Insights
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...
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.
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.
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Current Trends in the Healthcare Job Market
Vicki Salemi, Career Expert, discusses current trends in the healthcare job market and expectations for 2026. Recent data from the 2025 Monster Healthcare Market Report shows how the...
Efficacy of Pozelimab + Cemdisiran To Treat Paroxysmal Nocturnal Hemoglobinuria
Jun Ho Jang, MD, PhD, Professor, Division of Hematology-Oncology at Samsung Medical Center, discusses results from an open-label extension study testing pozelimab plus cemdisiran combination therapy...
FDA Approves Aqvesme (Mitapivat) for Treating Anemia in Alpha- and Beta-Thalassemia
The U.S. Food and Drug Administration (FDA) has approved Aqvesme (mitapivat) for the treatment of anemia in adults with alpha- or beta-thalassemia. Alpha- and beta-thalassemia are blood disorders...
Treatment With Elritercept for Patients With Myelofibrosis
Ciro Rinaldi, MD, Consultant Hematologist and Professor of Hematology at United Lincolnshire Hospital, discusses treatment with elritercept for patients with myelofibrosis (MF). MF is...
Duchenne Muscular Dystrophy (DMD) and Metachromatic Leukodystrophy (MLD) Recommended Uniform Screening Panel
The U.S. Department of Health and Human Services (HHS) has approved the addition of Duchenne muscular dystrophy (DMD) and metachromatic leukodystrophy (MLD) to the Recommended Uniform Screening...
Updated Phase 2 Results of Telquetamab + Teclistamab in Multiple Myeloma
Saad Usmani, MD, Myeloma Specialist and Cellular Therapist at Memorial Sloan Kettering Cancer Center, discusses updated results from the phase 2 RedirecTT-1 study of telquetamab + teclistamab in...
FDA Approves Obstructive Hypertrophic Cardiomyopathy Treatment
The U.S. Food and Drug Administration (FDA) has approved Myqorzo (aficamten) for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). oHCM is a rare genetic heart...
Results from the CARTITUDE-4 Clinical Trial in Multiple Myeloma
Binod Dhakal, MD, Associate Professor of Medicine at Medical College of Wisconsin, discusses results from the CARTITUDE-4 clinical trial in patients with multiple myeloma (MM). MM is a...
Results of Part 1a of the OLYMPIA-3 Study of Odronextamab+Chemotherapy in Diffuse Large B Cell Lymphoma
Jean-Marie Michot, MD, Institut Gustave Roussy, France, discusses results from Part 1A (dose escalation) of the OLYMPIA-3 study of odronextamab plus chemotherapy in patients with diffuse large...
2026 Orphan Drugs: PDUFA Dates and FDA Approvals
Below is the list of important regulatory dates for all orphan drugs for 2026. Prescription Drug User Fee Act (PDUFA) dates refer to deadlines for the FDA to review new drugs.2025...
Ibr+Ven Combination Therapy Versus Clb+Obi Combination Therapy in Chronic Lymphocytic Leukemia
Paolo Ghia, MD, PhD, Professor of Medical Oncology at the Università Vita-Salute San Raffaele in Milan, Italy, discusses data comparing ibrutinib plus venetoclax combination therapy with...
Results from the LINKER-MM4 Clinical Trial in Multiple Myeloma
Robert Orlowski, MD, PhD, Professor of Medicine at the University of Texas MD Anderson Cancer Center, discusses results from the LINKER-MM4 clinical trial testing Lynozyfic (linvoseltamab) to treat...
Current Status of Gene Therapies in Rare Neuromuscular Disorders
Current Status of Gene Therapies in Rare Neuromuscular Disorders Alan Beggs, PhD and Julie A Parsons, MDDrs. Beggs and Parsons discuss the current status of gene therapies in rare neuromuscular...
The cAMeLot-2 Trial Testing Bleximenib Combination Therapy for Acute Myeloid Leukemia
Elias Jabbour, MD, MD Anderson Cancer Center at the University of Texas, discusses the cAMeLot-2 study design of bleximenib combination therapy for patients with acute myeloid leukemia (AML). ...
Results of the BUTTERFLY Clinical Trial in Pediatric Patients With Dravet Syndrome
Joseph Sullivan, MD, Professor of Neurology and Pediatrics at the University of California at San Francisco, discusses the results of the BUTTERFLY clinical trial in pediatric patients with Dravet...
Current Status of Gene Therapy in Lysosomal Storage Disorders
Current Status of Gene Therapy in Lysosomal Storage Disorders Nicola Longo MD, PhD and Mark Roberts, MDIn this eight-part podcast series, Drs. Longo and Roberts discuss the current status of gene...
Phase 3 Data of Vonvendi for the Treatment of Von Willebrand Disease
Shayla Bergmann, MD, Pediatric Hematologist/Oncologist at the Medical University of South Carolina, discusses phase 3 data of Vonvendi for the treatment of Von Willebrand disease (VWD). ...
Broader Dosing Ranges of Skytrofa Now Available for Growth Hormone Deficiency
Lisa Abbott, MD, endocrinologist with Northern Nevada Endocrinology, discusses the expansion of broader dosing ranges of Skytrofa (lonapegsomatropin) now available for the replacement of endogenous...
FDA Approves Uplizna for the Treatment of Generalized Myasthenia Gravis
The U.S. Food and Drug Administration (FDA) has approved Uplizna (inebilizumab) for the treatment of generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) and...
Sotatercept for Pulmonary Arterial Hypertension Within the First Year After Diagnosis
A recent study, published in the New England Journal of Medicine, looked at the effects of sotatercept for pulmonary arterial hypertension (PAH) within the first year after diagnosis. PAH affects...
Reproduction and Pregnancy in Lysosomal Disorders
Best practices to manage pregnancy and other reproductive issues associated with lysosomal disorders (LDs)
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
CheckRare 5 hours ago