Clinical Insights
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). IgAN is characterized by the accumulation of IgA antibodies in the...
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 dystrophy type 1.
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 angioedema (HAE).
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 for affected families.
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FDA Approves Blenrep Combination Therapy for Multiple Myeloma
The U.S. Food and Drug Administration (FDA) has approved Blenrep (belantamab mafodotin) in combination with bortezomib and dexamethasone (BVd) for the treatment of adult patients with...
Early Results From the CHORD Clinical Trial in Otoferlin-Related Hearing Loss
Lawrence Lustig, MD, Professor at Columbia University Medical Center, discusses early results from the CHORD clinical trial testing gene therapy to treat otoferlin-related hearing loss. ...
FDA Approves Epioxa to Treat Keratoconus
The U.S. Food and Drug Administration (FDA) has approved Epioxa (riboflavin 5’-phosphate ophthalmic solution) for the treatment of patients with keratoconus. Keratoconus is a rare eye condition...
Lynch Syndrome: A Patient’s Experience With Genetic Testing and Increased Risk of Cancer
Tiffany Graham Charkosky, author and patient with Lynch syndrome, discusses her experience with genetic testing and her book LIVING PROOF: How Love Defied Genetic Legacy. Lynch...
FDA Approves Obinutuzumab for Patients With Lupus Nephritis
The U.S. Food and Drug Administration (FDA) has approved Gazyva/Gazyvaro (obinutuzumab) for the treatment of adults with active lupus nephritis who are receiving standard of care. In addition, the...
Cutaneous T-Cell Lymphoma
A Spotlight on Two Main Subtypes: Mycosis Fungoides and Sézary Syndrome Cutaneous T-cell lymphoma (CTCL) belongs to the non-Hodgkin lymphoma class of hematologic T-cell lymphoproliferative...
Ipsen’s Current Rare Disease Therapies: Approved and In Development
Christelle Huguet, PhD, Head of Research and Development at Ipsen, discusses the company’s current approved orphan drugs and those in development for rare diseases. Ipsen’s work in the...
New Data on the Use of Givinostat for Treatment of Patients With Duchenne Muscular Dystrophy
Scott Baver, PhD, Vice President of Medical Affairs at ITF Therapeutics, discusses new data on the use of Duvyzat (givinostat) for patients with Duchenne muscular dystrophy (DMD). DMD...
Diagnosis and Treatment of IgA Nephropathy
Jai Radhakrishnan, MD, Nephrologist and Professor at Columbia University Medical Center, discusses diagnosis and treatment of IgA nephropathy (IgAN). IgAN is an autoimmune kidney...
Neuroblastoma: Beat Childhood Cancer Research Consortium
Giselle Saulnier Sholler, MD, Division Chief for Pediatric Hematology/Oncology at Penn State University and Founder of the Beat Childhood Cancer Research Consortium, discusses the organization's...
Hemolytic Disease of the Fetus and Newborn: Outcomes of Intrauterine Transfusion and Patient Experiences
May Lee Tjoa, PhD, Senior Global Medical Affairs Leader: Nipocalimab and Maternal-Fetal Immunology at Johnson & Johnson, discusses data on hemolytic disease of the fetus and newborn (HDFN) from...
Rilzabrutinib Approval for Adult Patients With Immune Thrombocytopenia
Amit Mehta, MD, Medical Oncologist, discusses data that led to the approval of Wayrilz (rilzabrutinib) for adult patients with immune thrombocytopenia (ITP). ITP is a bleeding disorder...
Open-Label Extension Data of Del-Zota for Patients With Duchenne Muscular Dystrophy
Mike Flanagan, PhD, Chief Scientific Officer at Avidity Biosciences, discusses new data from the EXPLORE44 open-label extension of del-zota for treatment of patients with Duchenne muscular dystrophy...
FDA Approves Nerandomilast To Treat Patients With Idiopathic Pulmonary Fibrosis
The U.S. Food and Drug Administration (FDA) has approved Jascayd (nerandomilast) tablets for the treatment of patients with idiopathic pulmonary fibrosis (IPF). This is the first new therapy to be...
ISUOG World Congress 2025: Hemolytic Disease of the Fetus and Newborn
Jannine Williams, Compound Development Team Leader at Johnson & Johnson, discusses key takeaways from studies on hemolytic disease of the fetus and newborn (HDFN) presented at ISUOG World...
Neuroblastoma: New Approaches to Neuroblastoma Consortium
Araz Marachelian, MD, Pediatric Oncologist at Children’s Hospital Los Angeles, discusses the New Approaches to Neuroblastoma (NANT) Consortium and its work in neuroblastoma. ...
Results From the PEGASUS Clinical Trial of Pegvaliase in Patients With PKU
Kevin Eggan, PhD, Chief Scientific Officer at BioMarin, discusses new results from the PEGASUS clinical trial of Palynziq (pegvaliase) for treating patients with phenylketonuria (PKU). ...
Plans for Phase 2/3 Clinical Trial of Bexmarilimab Plus Standard of Care in Patients With Myelodysplastic Syndromes
Amer Zeidan, MBBS, MHS, Professor of Internal Medicine at Yale School of Medicine and Chief of the Division of Hematologic Malignancies at Yale Cancer Center, discusses plans for a phase 2/3...
Updates in Refractory Chronic Cough From ERS 2025
Jacky Smith, MB, ChB, FRCP, PhD, Chair of Respiratory Medicine at the University of Manchester, discusses topline results from the phase 2a RIVER trial for patients with refractory chronic cough...
Results From the LINKER-SMM1 Trial in Patients With High-Risk Smoldering Multiple Myeloma
Paula Rodriguez Otero, MD, PhD, Hematologist at the University of Navarra, discusses results from the LINKER-SMM1 trial in patients with high-risk smoldering multiple myeloma (HR-SMM). HR-SMM...
Reproduction and Pregnancy in Lysosomal Disorders
Best practices to manage pregnancy and other reproductive issues associated with lysosomal disorders (LDs)
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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.
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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 102 minutes ago