Clinical Insights
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). Steven...
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.
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 84-week results from the phase 3 OPTIMA trial extension, evaluating Pasatru (garetosmab-grts) in adults with fibrodysplasia ossificans progressiva (FOP).
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FDA Approves Lyrfigtu (Lirafugratinib) for the Treatment of Cholangiocarcinoma
The US Food and Drug Administration (FDA) has approved Lyrfigtu (lirafugratinib) for the treatment of cholangiocarcinoma with FGFR2 fusion or other rearrangement. Cholangiocarcinoma is a rare,...
Zilganersen for the Treatment of Alexander Disease
Amy Waldman, MD, pediatric neurologist and Medical Director of the Leukodystrophy Center at the Children’s Hospital of Philadelphia, discusses the approval of Zanvastro (zilganersen) to treat...
Phase 3 Results of Inhaled Treprostinil in Patients With Idiopathic Pulmonary Fibrosis
A study published in The New England Journal of Medicine examined results from the phase 3 TETON-1 clinical trial of inhaled treprostinil for idiopathic pulmonary fibrosis (IPF). IPF is a rare,...
KAT6 Syndromes
KAT6 syndromes are rare genetic neurodevelopmental disorders caused by genetic mutations in the KAT6A or KAT6B genes. KAT6 syndromes are characterized by speech and motor delay, low muscle tone,...
Revumenib + Chemotherapy in Newly Diagnosed AML With NPM1 Mutation: Phase 3 REVEAL-ND Study
My name is Dr. Joshua Zeidner, and I’m a Professor of Medicine and Chief of Leukemia Research at the University of North Carolina Lineberger Cancer Center. I’m here today to raise awareness around...
FDA Approves New Therapies for MPS IIIA and SMA | The CheckRare Brief Ep 8
This week we discuss the FDA's approval of Isembyld for SMA, the approval of a gene therapy for MPS IIIA, and the FDA's Center for Devices and Radiological Health new Rare Disease Impact...
Pulmonary Arterial Hypertension and Risk of Mental Health Comorbidities
A study published in Pulmonary Circulation evaluated the association between pulmonary arterial hypertension (PAH) diagnosis and risk of newly diagnosed mental health conditions. PAH is a rare...
Second Indication for Aqneursa in Ataxia Telangiectasia
The US Food and Drug Administration (FDA) has approved Aqneursa (levacetylleucine) for oral suspension to treat ataxia in patients with ataxia-telangiectasia who weigh at least 33 pounds (15 kg). ...
What to Expect: 2026 MGFA Scientific Session
Jenny McCue, Vice President of Global Research and Clinical Development at the Myasthenia Gravis Foundation of America (MGFA), discusses research highlights to be presented at the upcoming 2026 MGFA...
Topline Results From the SANRECO Trial Testing Divesiran in Polycythemia Vera
Marina Kremyanskaya, MD, PhD, Associate Professor at Icahn School of Medicine at Mount Sinai, discusses the phase 2 topline findings from the SANRECO clinical trial and the evolving polycythemia...
FDA Approves Gene Therapy (Fayuvi) for Pediatric Patients With Sanfilippo Syndrome (MPS IIIA)
The US Food and Drug Administration (FDA) has approved Fayuvi (rebisufligene etisparvovec-hopf; UX111) for the treatment of pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA;...
FDA Approves Generic Radioligand Therapy, Bexlutry, for Gastroenteropancreatic Neuroendocrine Tumors
The US Food and Drug Administration (FDA) has approved Bexlutry (lutetium Lu 177 dotatate) injection for the treatment of adults with somatostatin receptor-positive gastroenteropancreatic...
Results From the Voice of PBC Patient Survey
Carol Roberts, President of The PBCers Organization, discusses results from the Voice of PBC patient survey. PBC is a chronic, progressive autoimmune liver disease in which the bile...
Approval of Imaavy (Nipocalimab) and its Impact on Patients With Warm Autoimmune Hemolytic Anemia
Karen A. Jones, EdD, President and CEO of wAIHA Warriors, discusses the approval of Imaavy (nipocalimab) and its impact on patients with warm autoimmune hemolytic anemia (wAIHA). wAIHA is a...
Apitegromab Approved for Treatment of Patients With Spinal Muscular Atrophy
The US Food and Drug Administration (FDA) has approved Isembyld (apitegromab-mstn) for the treatment of spinal muscular atrophy (SMA) in patients ages 2 years and older who are currently receiving a...
First-Line Therapy Approved for Rare Lung Cancer | The CheckRare Brief Ep 7
This week, three developments highlight the rapidly changing rare disease treatment landscape: a Phase 3 setback in Angelman syndrome, continued competition in hereditary angioedema, and an...
Fabry Disease: Patients’ Real-World Problems and How to Manage Them
Fabry Disease: Patients' Real-World Problems and How to Manage Them Staci Kallish, DO; Dawn Laney, MS, CGS, CCRCThis educational program, hosted by Staci Kallish, DO, and Dawn Laney, MS, CGS, CCRC,...
Father Pursues Gene Therapy for Sons With Rare Genetic Variations of Duchenne Muscular Dystrophy
Tushar Tangsali, father to two sons with Duchenne muscular dystrophy (DMD), discusses how he’s working to develop a gene therapy for a unique genetic variation of DMD for his sons. DMD...
Fintepla Effectiveness and Safety in Patients With Lennox-Gastaut Syndrome
Amélie Lothe, PhD, Head of Global Medical Community of Rare Epilepsies at UCB, discusses results from a post hoc analysis of trajectories of Fintepla (fenfluramine) effectiveness and safety in...
New Huntington’s Disease Gene Therapy Application | The CheckRare Brief Ep 6
In this episode of The CheckRare Brief, we discuss two recent FDA approvals for rare diseases, including Zanvastro for Alexander disease and Besremi for essential thrombocythemia (ET). We...
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