Clinical Insights
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...
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.
3D Printed Treatment Options | The CheckRare Brief
In this episode, we discuss a new formulation of treatment for HAE, the FDA approval of Voceryq for recurrent respiratory papillomatosis, and 3D printed treatment options.
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FDA Approves Lumvoa (Veligrotug) for Thyroid Eye Disease
The US Food and Drug Administration (FDA) has approved Lumvoa (veligrotug) for the treatment of thyroid eye disease (TED), regardless of TED's duration or activity. TED is a chronic endocrine,...
Are All Cancers Rare Cancers? The Need for Better Classification Systems
Chadi Nabhan, MD, Hematologist and Oncologist, Chief Medical Officer at Ryght AI, and Vivek Subbiah, MD, Professor of Medicine at Stanford University and Executive Medical Director of Novel...
Spinal Muscular Atrophy: The Changing Definition of Success
Spinal muscular atrophy (SMA) has undergone a remarkable transformation over the past decade. Drs. Nancy Kuntz, Alicia Henriquez, and Angela Lek discuss how advances in disease-modifying therapies...
Congenital Adrenal Hyperplasia
Congenital adrenal hyperplasia (CAH) is a group of rare autosomal, recessive genetic disorders that impair production of cortisol (also known as hydrocortisone).[1,2] The hypothalmus and pituitary...
The Role of Rusfertide in Decreasing Phlebotomies in Patients With Polycythemia Vera
Andrew Kuykendall, MD, Clinical Researcher at Moffitt Cancer Center, discusses the role of rusfertide in decreasing phlebotomies in patients with polycythemia vera (PV). PV is a...
FDA Grants Full Approval to Afami-Cel for Patients With Synovial Sarcoma
The US Food and Drug Administration (FDA) has granted full approval to Tecelra (afamitresgene autoleucel; afami-cel) and expanded its indication to include patients ages 12 years and older with...
Results From the VISIONARY Clinical Trial of Sibeprenlimab in Patients With IgA Nephropathy
Vlado Perkovic, MD, Professor of Medicine and Provost at the University of New South Wales Sydney, discusses results from the VISIONARY clinical trial testing Voyxact (sibeprenlimab) in patients...
Long-Term Results of Ziftomenib Combination Therapy in Patients With Acute Myeloid Leukemia
Eunice S. Wang, MD, Chief of Leukemia at Roswell Park Comprehensive Cancer Center, discusses long-term results of ziftomenib in combination with venetoclax and azacitidine in patients with acute...
Oral Deucrictibant for On-Demand Treatment of Hereditary Angioedema Attacks
A study published in The Lancet Hematology evaluated the efficacy and safety of deucrictibant for the on-demand treatment of hereditary angioedema (HAE) attacks. Hereditary angioedema (HAE) is a...
Results From the CEPHEUS Clinical Trial of Daratumumab Combination Therapy in Newly Diagnosed Multiple Myeloma
Saad Usmani, MD, Hematologist-Oncologist at Memorial Sloan Kettering Cancer Center, discusses results from the CEPHEUS clinical trial of daratumumab combination therapy in patients with newly...
Effects of a SGLT2 inhibitor on Reducing Heart Failure in Carriers of Cardiomyopathy-Associated Genetic Variants
A study published in Nature Medicine evaluated whole-exome sequencing data from the DECLARE-TIMI 58 clinical trial to determine whether sodium–glucose cotransporter 2 (SGLT2) inhibition is...
Plasminogen Deficiency: Fibrin Accumulation and Its Effects on Patients
Plasminogen is a precursor protein that is converted into plasmin. Plasmin is crucial to break down fibrin, the main protein component of blood clots.[1] When the body does not produce sufficient...
Colors of SM: Expressions of Life with Systemic Mastocytosis
Fatima Scipione, Vice President of Global Patient Affairs at Blueprint Medicines, discusses the Colors of SM program. Systemic mastocytosis (SM) is a rare disease usually caused by...
The Importance of Early Diagnosis in IgA Nephropathy
Eric Lai, MD, Nephrologist at the West Coast Kidney Institute, discusses the importance of early diagnosis in IgA nephropathy (IgAN). IgAN is a kidney disorder that occurs when IgA...
Results From a Phase 3 Study of Voxzogo (Vosoritide) in Children With Hypochondroplasia
Andrew Dauber, MD, Chief of Endocrinology at Children’s National Hospital, discusses results from a phase 3 study of Voxzogo (vosoritide) in children with hypochondroplasia. ...
Results From the Phase 3 ElevAATe Clinical Trial in Alpha-1 Antitrypsin Deficiency
Alaa Hamed, MD, Global Head of Medical Affairs Rare Diseases at Sanofi, discusses results from the phase 2 ElevAATe clinical trial of efdoralprin alfa in patients with alpha-1 antitrypsin deficiency...
Immune Effector Cell-Associated Enterocolitis in Relapsed/Refractory Multiple Myeloma Treated With Ciltacabtagene Autoleucel
Yi Lin, MD, PhD, Hematologist/Oncologist at Mayo Clinic, discusses immune effector cell-associated enterocolitis (IEC-EC) in patients with relapsed/refractory multiple myeloma (R/R MM) treated with...
Medical Treatment and Stem-Cell Transplantation in Patients With Cutaneous T-Cell Lymphoma
Lauren Shea, MD, Assistant Professor of Hematology and Oncology, University of Alabama, Birmingham, discusses important aspects of medical treatment and stem-cell transplantation in patients with...
FDA Expands Approval of Hympavzi (Marstacimab) for Patients With Hemophilia
The US Food and Drug Administration (FDA) has approved an expanded indication for Hympavzi (marstacimab) to include treatment of patients with hemophilia A or B who are 12 years and older with...
Safety and Efficacy of Subcutaneous Efgartigimod PH20 in Ocular Myasthenia Gravis
Carolina Barnett-Tapia, MD, Neuromuscular Neurologist and the University of Toronto, discusses the safety and efficacy of subcutaneous efgartigimod PH20 in ocular myasthenia gravis (oMG). ...
Systemic Mastocytosis: Recognition, Diagnosis, and Clinical Management
Highlighting the early signs of Systemic Mastocytosis (SM), criteria and tools, and the importance of timely referral/testing
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TETON-2 Findings in Treatment of Idiopathic Pulmonary Fibrosis #podcast #raredisease
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Results of the Phase 3 OASIS Study of Oral Olorofim
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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
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
UCB Study Highlights the Burden of Ocular Myasthenia Gravis
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