Clinical Insights
FDA Approves Pimjetmi (Pimicotinib) for Patients With Tenosynovial Giant Cell Tumor
The US Food and Drug Administration (FDA) has approved Pimjetmi (pimicotinib) as a systemic treatment for adults with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection may cause worsening functional limitation or severe morbidity. TGCT is a...
UCB Study Highlights the Burden of Ocular Myasthenia Gravis
Anna Kole, Global Patient Engagement Lead for Myasthenia Gravis at UCB, discusses findings from a mixed-methods study examining the experiences and unmet needs of people living with ocular myasthenia gravis (oMG) and explains how patient perspectives could improve clinical research and care.
Highlights from The Liver Meeting 2026 | The CheckRare Brief
In this episode, we discuss highlights from The Liver Meeting, Ultragenyx program cuts, and ANI Pharmaceuticals’ offer to buy Egetis Therapeutics.
Inhaled Treprostinil Reduces Multiple Disease Progression Events in IPF
A post-hoc analysis of the phase 3 TETON-2 study, presented at the CHEST 2026 Annual Meeting in Phoenix, Arizona, found that inhaled treprostinil significantly reduced multiple disease progression events in patients with idiopathic pulmonary fibrosis (IPF).
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Myasthenia Gravis
Myasthenia gravis (MG) is a chronic autoimmune disease that disrupts communication between nerves and muscles, causing fluctuating weakness that often worsens with activity. It can affect the...
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...
Garetosmab Demonstrates Sustained Efficacy in Adults With Fibrodysplasia Ossificans Progressiva (FOP)
Richard Keen, MD, PhD, Director of the Metabolic Bone Disease Centre at the Royal National Orthopaedic Hospital in London and global principal investigator of the OPTIMA trial, discusses positive...
C3G and the Pediatric VALIANT Study
C3G and the Pediatric VALIANT Study Bradley Dixon, MDBradley Dixon, MD, a pediatric nephrologist, and Chief of Pediatric Nephrology, Children’s Hospital of Colorado, and Professor of Pediatrics,...
A Family’s Journey with Prader-Willi Syndrome
Maria Picone, co-founder and CEO of the TREND Community, and mother of a 14-year-old with Prader-Willi syndrome (PWS), discusses her family’s journey with PWS, including why more research and better...
3D Printed Treatment Options | The CheckRare Brief
In this episode, we discuss a new formulation of treatment for HAE, the FDA approval of Voceryq for recurrent respiratory papillomatosis, and 3D printed treatment options. New...
Molecular Changes Associated With Teprotumumab Treatment in Thyroid Eye Disease
Sarah Cheng, MD, PhD, a clinical oculoplastic fellow at the University of California, San Diego, discusses findings from an exploratory study examining the biological changes in orbital tissue...
FDA Grants Accelerated Approval to Voceryq for Recurrent Respiratory Papillomatosis
The US Food and Drug Administration (FDA) has granted accelerated approval to Voceryq (doruxapapogene ralaplasmid and lalikinogene sifuplasmid-gxfd), previously referred to as INO-3107, to reduce...
Mirum’s Livmarli Achieves Primary Endpoint in Phase 3 EXPAND Study
Mirum Pharmaceuticals recently announced positive topline results from its phase 3 EXPAND study, evaluating Livmarli (maralixibat) for cholestatic pruritus in patients six months and older with...
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...
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...
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...
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...
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...
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...
New Formulation of Berotralstat Approved for Adults and Adolescents With Hereditary Angioedema
The US Food and Drug Administration (FDA) has approved the oral pellet formulation of once-daily Orladeyo (berotralstat) for prophylactic therapy in patients ages 12 years and older with hereditary...
FDA Approves Gazyva to Reduce Relapse Risk in Patients With Idiopathic Nephrotic Syndrome
Andrea Callaway, president and founder of The Nephrotic Syndrome Foundation, discusses the FDA approval of Gazyva (obinutuzumab) for idiopathic nephrotic syndrome (INS) and what the approval means...
FDA Approves New Formulation of Sjögren’s Disease Treatment
The US Food and Drug Administration (FDA) has approved Evoxac (cevimeline HCI) oral solution to address dry mouth in patients with Sjögren’s disease (SjD). SjD is an autoimmune disorder that largely...
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...
Organoids and Lab-Grown Models in Lysosomal Disorders
Mia Horowitz, PhD, Aitor Aguirre, PhD, and Ying Sun, PhD, discuss the use of organoid models in lysosomal disorder research and drug development.
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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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Anna Kole, Global Patient Engagement Lead for Myasthenia Gravis at UCB, discusses the findings from a mixed-methods study examining the experiences and unmet needs of people living with ocular myasthenia gravis (oMG) and explains how patient perspectives could improve clinical research and Read morecare.
View the full interview at https://checkrare.com/ucb-study-highlights-the-burden-of-ocular-myasthenia-gravis/
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
UCB Study Highlights the Burden of Ocular Myasthenia Gravis
CheckRare 12 hours ago