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 Jideytro (Zidesamtinib) for Adults With ROS1-Positive Non-Small Cell Lung Cancer
The US Food and Drug Administration (FDA) has approved Jideytro (zidesamtinib) for the treatment of adult patients with locally advanced or metastatic ROS1-positive non-small cell lung cancer...
Results From the PROGRESS Study of Zilurgisertib in Patients With Fibrodysplasia Ossificans Progressiva
Joanne Quan, MD, Chief Medical Officer of Mirum Pharmaceuticals, discusses results from the PROGRESS study of zilurgisertib in patients with fibrodysplasia ossificans progressiva (FOP). FOP...
Investigating Results From the MAVORIC Trial in Patients With CTCL
H. Miles Prince, MD, MBBS, Professor at the University of Melbourne, Australia, and Director, Peter MacCallum Cancer Center, and Director of Molecular Oncology and Cancer Immunology, Epworth...
DAHLIAS Clinical Trial Evaluating Nipocalimab in Sjögren’s Disease
Robert Hal Scofield, MD, Professor of Medicine, at the University of Oklahoma and Oklahoma Medical Research Foundation, discusses the DAHLIAS clinical trial evaluating nipocalimab in Sjögren’s...
Mogamulizumab in Danish Patients With CTCL
Lena Specht, MD, DMSc, Professor of Clinical Oncology, and Chief Oncologist at Rigshopitalet, University of Copenhagen, Denmark, discusses her Danish study supporting the use of mogamulizumab in...
2-Year Data on Crinecerfont for the Treatment of Patients With Congenital Adrenal Hyperplasia
Oksana Hamidi, DO, Associate Professor at the University of Texas Southwestern Medical Center, discusses 2-year data on crinecerfont for the treatment of patients with congenital adrenal hyperplasia...
Increased Dosing Interval of Mogamulizumab in Patients With CTCL
Christiane Querfeld, MD, PhD, dermatologist and and Director, Multidisciplinary Cutaneous Lymphoma Program, Professor at the City of Hope National Medical Center and the Beckman Research Institute,...
Lipodystrophy Research Presented at ENDO 2026
Elif Oral, MD, Professor at the University of Michigan, discusses advances in lipodystrophy research presented at ENDO 2026. Lipodystrophies are rare metabolic disorders characterized...
Prader-Willi Syndrome: Diazoxide Choline Extended Release Improves Hyperphagia
Evelien Gevers, PhD, MD, Consultant Pediatrician and Reader in Endocrinology and Diabetes at Barts Health NHS Trust, discusses effects of diazoxide choline extended release (DCCR) on hyperphagia in...
The Importance of Patient and Physician Communication in the Care of Hypoparathyroidism
Katie Gillick, Patient Advocate at Orsini Specialty Pharmacy, discusses the importance of patient and physician communication in the care of hypoparathyroidism. Hypoparathyroidism is a...
Primary IGF-1 Deficiency: Causes, Early Detection, and Treatment
Primary IGF-1 Deficiency Robert Rapaport, MD, Professor of Pediatric Endocrinology, and Director of the Comprehensive Growth Center at the Icahn School of Medicine, Mount Sinai Medical Center, New...
FDA Approves Tregzi for Treatment of Graft-Versus-Host Disease in Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
The US Food and Drug Administration (FDA) has approved Orca-T (Tregzi), the first regulatory T cell-based immunotherapy for improving chronic graft-versus-host disease (GVHD)-free survival in adult...
Congenital Adrenal Hyperplasia: Effect of Crenessity (Crinecerfont) on Bone Age Advancement
Maria Vogiatzi, MD, Division of Endocrinology at the Children's Hospital of Philadelphia, discusses the effect of Crenessity (crinecerfont) on bone age advancement in patients with congenital...
FDA Grants Accelerated Approval to Atacicept for the Treatment of IgA Nephropathy
The US Food and Drug Administration (FDA) has granted accelerated approval to Trutakna (atacicept) for the treatment of adults with primary immunoglobulin A nephropathy (IgAN) at risk for disease...
Congenital Hyperinsulinism Diagnosis and Management
Kristen Rohli, PhD, Associate Director of Research at Congenital Hyperinsulinism International, discusses congenital hyperinsulinism (HI) diagnosis and management. Congenital HI is a...
Results from the CALIBRATE Clinical Trial in Patients With Autosomal Dominant Hypocalcemia Type 1
Rachel Gafni, MD, Senior Research Physician at the National Institutes of Health, discusses results from the CALIBRATE clinical trial in patients with autosomal dominant hypocalcemia type 1 (ADH1)....
A Patient’s Diagnostic Journey With Congenital Adrenal Hyperplasia
Melanie Gander, mother of a son with congenital adrenal hyperplasia (CAH), and Ambassador with Neurocrine Biosciences, discusses her son’s diagnostic journey with congenital adrenal hyperplasia....
Mental Health Challenges and Care Gaps in Patients With Lipodystrophy
Kate Stratton, Executive Director of Lipodystrophy United, discusses mental health challenges and care gaps in patients with lipodystrophy. Lipodystrophies are rare metabolic disorders...
Treatment With Xywav (Low Sodium Oxybate) in Patients With Narcolepsy and Idiopathic Hypersomnia
Logan Schneider, MD, Adjunct Clinical Associate Professor of Psychiatry and Behavioral Sciences at Stanford University, discusses treatment with Xywav (low sodium oxybate) in patients with sleep...
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,...
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