Clinical Insights
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).
C3G and the Pediatric VALIANT Study
Bradley Dixon, MD, a pediatric nephrologist, and Chief of Pediatric Nephrology, Children’s Hospital of Colorado, and Professor of Pediatrics, University of Colorado School of Medicine, discusses complement 3 glomerulopathy (C3G) and the pediatric VALIANT study.
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 data are so important.
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FDA Approves Multiple Myeloma Treatment, Denies Neuroendocrine Tumors Treatment
On this week’s episode of The CheckRare Brief, we discuss FDA’s approval of Zenbexus (iberdomide) to treat patients with multiple myeloma, the complete response letter issued to ITM-11 to...
Importance of Patient Listening in The Development of Rare Disease Treatments
Matt Trudeau, President of ITF Therapeutics, discusses the importance of patient listening in the development of Duvyzat (givinostat) for Duchenne muscular dystrophy (DMD). DMD is a...
Rare Endocrine Exchange: Why This Conversation Matters
Rare endocrine disorders can be difficult to recognize, challenging to diagnose, and even harder for patients to navigate. For many patients, the journey to an accurate diagnosis can take years....
FDA Approves Iberdomide Combination Therapy for Relapsed/Refractory Multiple Myeloma
The US Food and Drug Administration (FDA) has granted accelerated approval to iberdomide (Zenbexus) in combination with daratumumab and hyaluronidase-fihj and dexamethasone for adults with multiple...
Topline Results from the MonumenTAL-6 Clinical Trial in Adults With Relapsed/Refractory Multiple Myeloma
Ajay K. Nooka, MD, MPH, FACP, Director of the Myeloma Program at the Emory University School of Medicine, discusses topline results from the MonumenTAL-6 clinical trial testing Tecvayli...
Follow-Up Data of Safusidenib in Patients With IDH-Mutant Gliomas
Alexandra Miller, MD, PhD, Chief of Neuro-Oncology and Co-Director of the Brain and Spine Tumor Center at the Perlmutter Cancer Center at NYU Langone Health, discusses follow-up data on the safety...
FDA Approves New Narcolepsy Treatment | The CheckRare Brief
On this week’s episode of The CheckRare Brief, we discuss FDA's approval of Orzeyful (oveporexton) to treat patients with narcolepsy type 1, CAMP4’s first-in-human clinical trial for patients...
ATLAS-OLE Subgroup Analysis of Fitusiran in Patients With Hemophilia
Guy Young, MD, Director of the Hemostasis and Thrombosis Program at Children's Hospital Los Angeles, discusses an ATLAS-OLE subgroup analysis of fitusiran in patients with hemophilia. ...
Safety and Efficacy of Kygevvi in Thymidine Kinase 2 Deficiency
Caterina Garone, PhD, MD, Associate Professor at the University of Bologna, discusses safety and efficacy data on Kygevvi (doxecitine and doxribtimine) in patients with thymidine kinase 2 deficiency...
Now Enrolling: GALILEO-3 Trial Evaluating FLT201 for Adults With Gaucher Disease Type 1
Priya Kishnani, MD, Professor and Chief of the Division of Medical Genetics at Duke University, discusses the GALILEO-3 clinical trial of FLT201 in adults with Gaucher disease type 1 (GD1). ...
FDA Advisory Board’s Concerns About New Duchenne Drug, Brain Fog in Immune Thrombocytopenia
The CheckRare Brief delivers a concise summary of the week's most important developments in rare diseases. Each episode covers FDA approvals, clinical trial updates, conference highlights,...
FDA Approves Orzeyful (Oveporexton) for the Treatment of Adults With Narcolepsy Type 1
The US Food and Drug Administration (FDA) has approved Orzeyful (oveporexton) for the treatment of adults with narcolepsy type 1 Narcolepsy is a chronic neurological disorder characterized by an...
LOTUS Study in Patients With Rett Syndrome Treated With Daybue (Trofinetide)
Ryan Bucco, PharmD, Vice President of Medical Affairs in Rare Disease at Acadia, discusses the LOTUS study in patients with Rett syndrome treated with Daybue (trofinetide). Rett syndrome is a...
Results From the ENERGY Clinical Trial Testing Imaavy (Nipocalimab) in Warm Autoimmune Hemolytic Anemia (wAIHA)
Bruno Fattizzo, MD, Department of Oncology and Hemato-Oncology at the University of Milan, discusses results from the ENERGY clinical trial of Imaavy (nipocalimab) in warm autoimmune hemolytic...
Initiation of Phase 3 FORZETTO Clinical Trial Testing Zeleciment Rostudirsen in Duchenne Muscular Dystrophy
Doug Kerr, MD, PhD, MBA, Chief Medical Officer of Dyne Therapeutics, discusses the initiation of the phase 3 FORZETTO trial of zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251), in...
August Is SMA Awareness Month
August is SMA Awareness Month, a time dedicated to increasing understanding of spinal muscular atrophy (SMA) and supporting the individuals and families impacted by this rare genetic neuromuscular...
Phase 2a GATEWAY Trial of PORT-77 in Patients With Erythropoietic Protoporphyria Shows Promise
Pete Schmidt, MD, MSc, Chief Medical Officer of Portal Therapeutics, discusses data from the phase 2a GATEWAY trial testingPORT-77, an ABCG2 inhibitor, in patients with erythropoietic protoporphyria...
Patient Perspective: Diagnosis and Treatment Journey With ROS1-Positive Non-Small Cell Lung Cancer
Jim Herbert, a patient with ROS1-positive non-small cell lung cancer (NSCLC), discusses his diagnosis and treatment journey. ROS1-positive NSCLC is a rare and aggressive lung cancer...
Results from the MajesTEC-9 and CARTITUDE-4 Clinical Trials in Patients With Relapsed/Refractory Multiple Myeloma
Roberto Mina, MD, Assistant Professor at Winship Cancer Institute at Emory University, discusses results from the MajesTEC-9 and CARTITUDE-4 clinical trials in patients with relapsed/refractory...
Going Beyond Lab Values: Cognitive Impairment in Patients With Immune Thrombocytopenia
David Kuter, MD, DPhil, Director of Clinical Hematology at Massachusetts General Hospital and Professor of Medicine at Harvard Medical School, discusses cognitive impairment in patients with immune...
Neuroblastoma
Neuroblastoma is a rare childhood cancer characterized by a neuroendocrine tumor originating in neuroblasts or neural crest progenitor cells.
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