Clinical Insights
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...
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 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).
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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...
Zopapogene Imadenovec in Treating Patients With Recurrent Respiratory Papillomatosis
Clint Allen, MD, Senior Investigator, and Scott Norberg, DO, Associate Research Physician at Center for Cancer Research at the National Cancer Institute, discuss results from a study testing the...
Patient Perspective: TUBB4B and The Need for Awareness
Makayla Alger, patient advocate with TUBB4B, and her mother Joann Alger, discuss their experience with the condition and the need for awareness. The TUBB4B gene is a gene believed to...
FDA Extends Evinacumab Indication to Include Patients With HoFH Ages 1 to 5 Years
The U.S. Food and Drug Administration (FDA) has approved Evkeeza (evinacumab-dgnb) for the treatment of children ages 1 to 5 years old with homozygous familial hypercholesterolemia (HoFH),...
Biomarker Validation in Niemann-Pick Disease Type C
Krista Casazza, PhD, co-author of the recent article “Biomarker Validation in NPC1: Foundations for Clinical Trials and Regulatory Alignment” in the Journal of Inherited and Metabolic Disease,...
Neuroblastoma: The Children’s Oncology Group
Navin Pinto, MD, Professor of Pediatrics at the University of Colorado Anschutz Medical Campus and Colorado Children’s Hospital, discusses the Children’s Oncology Group and their work in...
FDA Approves Paltusotine for Treatment of Adults With Acromegaly
The U.S. Food and Drug Administration (FDA) has approved Palsonify (paltusotine) for the first-line treatment of adults with acromegaly with inadequate response to surgery and/or for whom surgery is...
Sophie’s Hope Foundation: A GSD1b Patient Advocacy Organization
Jamas LaFreniere, Founder of Sophie’s Hope Foundation and CURE GSD1b, and Blair Stone-Schneider, Executive Director of Sophie’s Hope Foundation, discuss their patient advocacy organization and...
Treating NF1-PN With Mirdametinib
Phioanh Leia Nghiemphu, MD, Professor of Clinical Neurology at University of California Los Angeles, discusses the use of mirdametinib for neurofibromatosis type 1 with symptomatic plexiform...
Treating Rare Lung Cancer (ROS-1 Positive NSCLC) With Taletrectinib
Geoffrey Liu, MD, Senior Scientist at the Princess Margaret Cancer Centre at the University of Toronto, discusses new data on the treatment of ROS1-positive non-small cell lung cancer (NSCLC) with...
Long-Term Safety and Efficacy Data on Givinostat for Patients With Duchenne Muscular Dystrophy
Scott Baver, PhD, Vice President of Medical Affairs at ITF Therapeutics, discusses long-term safety and efficacy data on givinostat for patients with Duchenne muscular dystrophy (DMD). ...
FDA Approves Elamipretide for Patients With Barth Syndrome
The U.S. Food and Drug Administration (FDA) has granted accelerated approval to Forzinity (elamipretide HCI) for the treatment of patients with Barth syndrome. The treatment is indicated to improve...
New Staging Tool for Cutaneous T-cell Lymphoma (PROBEinCTCL)
Greg Palko, Vice President and Oncology Franchise Head of Kyowa Kirin North America, discusses a new cutaneous T-cell lymphoma (CTCL) Staging Tool. CTCL is a rare group of malignancies...
Diagnosis and Management of Hypoparathyroidism
Michele Rayes, Associate Director of the HypoPARAthyroidism Association and patient, discusses the diagnosis and management of hypoparathyroidism. Hypoparathyroidism is a rare endocrine...
Patient Perspective: Lipodystrophy Diagnostic Journey
Sharon Halperin, Research Director for Lipodystrophy United and patient, discusses her diagnostic journey with lipodystrophy. Lipodystrophies are rare metabolic disorders characterized...
Daily Symptom Burden of Hypoparathyroidism
Patty Keating, Executive Director of the HypoPARAthyroidism Association and patient, discusses the daily symptom burden of hypoparathyroidism. Hypoparathyroidism is a rare endocrine disorder...
CAHtalyst Clinical Trials in Adults With Congenital Adrenal Hyperplasia
Vivian Lin, MD, Executive Medical Director of Medical Affairs at Neurocrine, discusses the CAHtalyst clinical trials in adults with congenital adrenal hyperplasia (CAH). CAH refers to...
Unmet Needs of Patients With Cushing’s Syndrome
Alessandro Albuquerque, MD, PhD, Chief Medical Officer of Recordati Rare Diseases North America, discusses unmet needs of patients with Cushing’s syndrome. Cushing's syndrome is a rare...
Trends and Social Determinants of Teprotumumab in Thyroid Eye Disease
Jui-En Lo, MD, Resident of Internal Medicine at MetroHealth, discusses trends and social determinants of teprotumumab in thyroid eye disease (TED). TED is a rare autoimmune disease...
Investigating Results From the MAVORIC Trial in Patients With CTCL
H. Miles Prince, MD, MBBS, describes results of an investigation of previous results from the MAVORIC trial in patients with CTCL.
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Highlights from The Liver Meeting 2026 | The CheckRare Brief
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New research is helping advance the understanding and treatment of rare kidney diseases.
The VALIANT study evaluated pegcetacoplan, a targeted complement therapy, in patients with C3G and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN).
Explore the study Read moreand hear more about the importance of advancing treatment options for patients, including children.
Read the full story: https://checkrare.com/c3g-and-the-pediatric-valiant-study/
The Need for Earlier Pulmonary Hypertension Screening in Interstitial Lung Disease
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/
#MyastheniaGravis #RareDiseaseEducation #HealthcareProfessionals #ClinicalResearch #PatientEducation
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
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
Interim PHINDER Analyses in Interstitial Lung Disease
CheckRare 18 minutes ago