Clinical Insights
Myasthenia Gravis
Myasthenia gravis (MG) is a chronic autoimmune disease that disrupts communication between nerves and muscles, causing fluctuating weakness that often worsens with activity. It can affect the muscles controlling the eyes, face, speech, swallowing, limbs, and...
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.
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Decoding Delays, Connecting the Care: The Challenge of Diagnosing Rare Endocrine Disorders
For people living with rare endocrine disorders, getting an accurate diagnosis can be a long and frustrating journey. Symptoms may be subtle, overlap with more common conditions, or involve...
FDA Approves First Treatment for Alexander Disease
The US Food and Drug Administration (FDA) has approved Zanvastro (zilganersen) for the treatment of pediatric and adult patients with Alexander disease. Alexander disease is a rare leukodystrophy...
Data on Maribavir in Post-Transplant Cytomegalovirus
Neil Inhaber, MD, FRCPC, Therapeutic Area Head of Global Medical, Rare Disease at Takeda, discusses data on maribavir in post-transplant cytomegalovirus (CMV) presented at the American Transplant...
New Graves’ Disease Community Website
Christine Gustafson, Executive Director and CEO of the TED Community Organization, discusses the new Graves’ Community website. Graves’ disease is an autoimmune disease characterized...
Narcolepsy Diagnosis and Symptoms: Understanding the Daily Burden of the Disease
Tammy Anderson, Executive Director, and Jenny Rose, Manager of Outreach and Management at Wake Up Narcolepsy, discuss the challenges of diagnosing narcolepsy and the profound impact the disorder can...
FDA Approves Rasonque (daraxonrasib) for Adults With Metastatic Pancreatic Cancer
The US Food and Drug Administration (FDA) has approved Rasonque (daraxonrasib) for the treatment of adults with metastatic pancreatic adenocarcinoma (PDAC) who have received at least one prior...
New FDA Approvals for Rare Diseases: From Autoimmune Disorders to Pancreatic Cancer | The CheckRare Brief Ep 5
In this episode of The CheckRare Brief, we discuss four recent FDA approvals, including LISRAYA for dermatomyositis and Imaavy for warm autoimmune hemolytic anemia (wAIHA), Mimrylo for...
FDA Approves Rusfertide for Treatment of Adults With Polycythemia Vera
The US Food and Drug Administration (FDA) has approved Mimrylo (rusfertide) for the treatment of adults with polycythemia vera (PV). PV is a condition characterized by an increased production of red...
FDA Approves Besremi for Treatment of Adults With Essential Thrombocythemia
The US Food and Drug Administration (FDA) has approved Besremi (ropeginterferon alfa-2b-njft) for the treatment of adults with essential thrombocythemia (ET). ET is a condition characterized by an...
Narcolepsy Awareness Month
September is Narcolepsy Awareness Month, a time dedicated to increasing understanding of narcolepsy and supporting the individuals and families impacted by this rare neurological disorder. ...
FDA Approves First Treatment for Dermatomyositis
The US Food and Drug Administration (FDA) has approved Lisraya (brepocitinib) 30 mg for the treatment of adults with dermatomyositis (DM). DM is a rare autoimmune condition that targets small blood...
Kleefstra Syndrome: Development of RNA Amplifier Therapy
Eric Scheeff, PhD, Chief Scientific Officer at iDefine, discusses the treatment development of RNA amplifiers in patients with Kleefstra syndrome. Kleefstra syndrome is a rare disorder...
Predicting Treatment Response Outcomes With Proteomic and Machine Learning Analyses in Myasthenia Gravis
Henry Kaminski, MD, Department of Neurology, and Linda Kusner, PhD, Department of Pharmacology & Physiology at George Washington University, discuss predicting response outcomes with proteomic...
Making Travel Accessible: SMAshing My Limits Partners With Becoming rentABLE
Tracey Dawson, PhD, Senior Vice President, US Therapeutic Area Head Neuroscience at Novartis, discusses the collaboration between Novartis, SMAshing My Limits, and Becoming rentABLE, striving to...
Getting a Rare Disease Drug Approved Is Only Half the Battle | The CheckRare Brief Ep 4
FDA approval is a major milestone, but for patients with rare diseases, it is only the beginning. In this episode of The CheckRare Brief, we look at two new rare disease approvals, ongoing...
FDA Approves First Treatment for Warm Autoimmune Hemolytic Anemia (wAIHA)
The US Food and Drug Administration (FDA) has approved Imaavy (nipocalimab) for the treatment of patients ages 12 years and older with warm autoimmune hemolytic anemia (wAIHA) who are currently or...
Addressing Patient Experiences With IgA Nephropathy
Gaia Coppock, MD, Nephrologist and Associate Professor of Clinical Medicine at the University of Pennsylvania, discusses patient experiences with IgA nephropathy (IgAN). IgAN is...
FDA Approves Pasatru (Garetosmab) for Adults With Fibrodysplasia Ossificans Progressiva
The US Food and Drug Administration (FDA) has approved Pasatru (garetosmab-grts) to reduce formation of new heterotopic ossification (HO) lesions and clinician-assessed flare-ups in adults with...
FDA Grants Accelerated Approval to AAV8-Based Gene Therapy (Genglycos) for Patients With Glycogen Storage Disease Type 1a
The US Food and Drug Administration (FDA) has granted accelerated approval for Genglycos (pariglasgene brecaparvovec-opnr), also known as DTX401, in patients eight years and older with glycogen...
Patient Perspective: Diagnostic Journey With IgA Nephropathy
Byron Wade, a patient with IgA nephropathy (IgAN), discusses his personal diagnostic journey and a recent study looking at other patients’ experiences. IgAN is characterized by the...
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