Clinical Insights
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. Ultragenyx Ends Angelman Syndrome and Wilson Disease Programs Ultragenyx announced this week that it's...
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).
Myasthenia Gravis
Myasthenia gravis is a chronic autoimmune neuromuscular disease characterized by weakness of the skeletal muscles. Common symptoms include weakness of the muscles that control the eye and eyelid, facial expressions, chewing, talking, and swallowing.
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.
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Efficacy of Vosoritide to Treat Hypochondroplasia
Andrew Dauber, MD, MMsc, Chief of Endocrinology at Children’s National, discusses the efficacy of vosoritide to treat persons with hypochondroplasia. Hypochondroplasia is a rare...
Efficacy of Infigratinib on Achondroplasia
Daniela Rogoff, MD, PhD, Chief Medical Officer, Skeletal Dysplasia of BridgeBio, discusses the efficacy of infigratinib to treat persons with achondroplasia. Achondroplasia is a disorder of...
FDA Grants Breakthrough Therapy Designation to Cimzia for Pregnancy Complications in Antiphospholipid Syndrome
Jane Salmon, MD, Professor of Medicine at Weill Cornell Medical College, discusses antiphospholipid syndrome (APS), its impact on pregnancy, and emerging research into Cimzia (certolizumab pegol) as...
New Stem Cell Method Could Advance Research Into Spinal Muscular Atrophy
Kyle Loh, PhD, Associate Professor at Stanford Medicine, discusses research that could help scientists better understand spinal muscular atrophy (SMA) by generating new motor neurons from human...
Ultragenyx Sells PRV for $210M | The CheckRare Brief Ep 11
In this episode, we discuss Ultragenyx’s Priority Review Voucher that sold for $210 million, the efficacy of newborn screening on MLD, and ethical considerations needed to design and conduct...
FDA Approves Gazyva for Idiopathic Nephrotic Syndrome, Offering a New Treatment Option
Keisha L. Gibson, MD, MPH, pediatric nephrologist at the UNC School of Medicine, discusses the approval of Gazyva (obinutuzumab) for patients with childhood-onset idiopathic nephrotic syndrome...
FDA Approves Jaypirca (Pirtobrutinib) for Two Rare Blood Cancers
The US Food and Drug Administration (FDA) has approved pirtobrutinib for adults with previously untreated chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) with no known 17p...
Taletrectinib for ROS1-Positive Non-Small Cell Lung Cancer
Jorge Nieva, MD, Associate Professor at the Keck School of Medicine, discusses new analyses from the TRUST studies evaluating taletrectinib in patients with ROS1-positive non-small cell lung cancer...
Plasma Proteomics May Predict Disease Manifestations in ALS
Michael Benatar, MD, PhD, Department of Neurology and ALS Center, University of Miami Miller School of Medicine, discusses the use of plasma proteomics in predicting clinical manifestations of...
Therapies for MCT8 Deficiency and Nephrotic Syndrome Get FDA Approval | The CheckRare Brief Ep 10
On September 28th, the FDA approved Emcitate, or tiratricol, from Egetis Therapeutics. It's the first-ever treatment for MCT8 deficiency, a condition also known as Allan-Herndon-Dudley syndrome. ...
Priority Review for Dersimelagon in the Treatment of Erythropoietic Protoporphyria and X-Linked Protoporphyria
Lukasz Jarzyna, Vice President of Rare Disease at LEO Pharma, discusses the Priority Review for dersimelagon in the treatment of erythropoietic protoporphyria (EPP) and X-linked protoporphyria...
A Patient’s Journey With Essential Thrombocythemia and Polycythemia Vera
Nona Baker, Co-Chair of MPN Voice and patient, discusses her journey with essential thrombocythemia (ET) and polycythemia vera (PV), including diagnosis and treatment. ET is a condition...
FDA Approves Camzyos (Mavacamten) for Obstructive Hypertrophic Cardiomyopathy
The US Food and Drug Administration (FDA) has approved Camzyos (mavacamten) for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and...
FDA CDER’s Accelerating Rare Disease Cures Program
Paz Vellanki, MD, PhD, Vice President of Clinical Development at Precision for Medicine, discusses the FDA CDER’s Accelerating Rare Disease Cures (ARC) Program. The ARC Program was launched...
Rett Syndrome Awareness Month
October is Rett Syndrome Awareness Month. Rett syndrome is a neurodevelopmental condition that primarily affects girls. People with the disease appear to have normal psychomotor development during...
FDA Approves Gazyva (Obinutuzumab) for Treatment of Childhood-Onset Idiopathic Nephrotic Syndrome
The US Food and Drug Administration (FDA) has approved Gazyva (obinutuzumab) to reduce the risk of relapse in adult and pediatric patients 2 years of age and older with frequently relapsing or...
FDA Approves First Treatment for MCT8 Deficiency
The US Food and Drug Administration (FDA) has approved Emcitate (tiratricol) for the treatment of peripheral thyrotoxicosis in patients with monocarboxylate transporter 8 (MCT8) deficiency. MCT8...
Safety and Efficacy of Ralinepag in Patients With Pulmonary Arterial Hypertension
A study published in The Lancet evaluated the safety and efficacy of ralinepag in patients with pulmonary arterial hypertension (PAH). PAH is a rare condition affecting the heart and lungs,...
FDA Approves Atebrioz (zilurgisertib) for Treatment of Fibrodysplasia Ossificans Progressiva
The US Food and Drug Administration (FDA) has approved Atebrioz (zilurgisertib) tablets to reduce the volume of total new heterotopic ossification (HO) in patients ages 12 years and older with...
New Rare Disease Center of Excellence and FDA Advances in Rare Disease | The CheckRare Brief Ep 9
This week on The CheckRare Brief, we are joined by Eugene Lee, CMI Media Group’s Group President, Specialty Communications, for an industry perspective on some of the biggest developments in rare...
Cutaneous T-Cell Lymphoma
Cutaneous T-cell lymphoma (CTCL) belongs to the non-Hodgkin lymphoma class of hematologic T-cell lymphoproliferative disorders.
Recent Videos
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
Phase 3 OASIS Study Shows Promise of Oral Olorofim for Invasive Aspergillosis
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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
Phase 3 OASIS Study Shows Promise of Oral Olorofim for Invasive Aspergillosis
🧬 A new chapter in gene therapy for spinal muscular atrophy (SMA).
How is Itvisma changing the treatment landscape for people living with SMA? In this CheckRare feature, John Day, MD, PhD, of Stanford University, discusses the development of this one-time gene therapy, its targeted Read moredelivery approach, and clinical trial findings on motor function.
Discover what this advancement could mean for the future of SMA treatment.
🔗 Read the full article: https://checkrare.com/itvisma-gene-therapy-for-patients-with-spinal-muscular-atrophy/
#CheckRare #SpinalMuscularAtrophy #SMA #RareDisease #GeneTherapy #MedicalInnovation
🧬 A new chapter in gene therapy for spinal muscular atrophy (SMA).
How is Itvisma changing the treatment landscape for people living with SMA? In this CheckRare feature, John Day, MD, PhD, of Stanford University, discusses the development of this one-time gene therapy, its
TETON-2 Findings in Treatment of Idiopathic Pulmonary Fibrosis #podcast #raredisease
CheckRare 22 hours ago