Clinical Insights
C3G and the Pediatric VALIANT Study
C3G and the Pediatric VALIANT Study Bradley Dixon, MDBradley 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...
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.
Molecular Changes Associated With Teprotumumab Treatment in Thyroid Eye Disease
Sarah Cheng, MD, PhD, a clinical oculoplastic fellow at the University of California, San Diego, discusses findings from an exploratory study examining the biological changes in orbital tissue associated with Tepezza (teprotumumab-trbw) treatment. The findings were presented at the 2026 American Academy of Ophthalmology (AAO) Annual Meeting in New Orleans, Louisiana.
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Impact of Vyvgart (Efgartigimod Alfa) Approval for Patients with Seronegative Myasthenia Gravis
Jeff Guptill, MD, Neuromuscular Clinical Development Lead at Argenx, discusses the impact of the Vyvgart (efgartigimod alfa) approval on the myasthenia gravis (MG) community to treat patients with...
Preferences and Perceptions of Acute Seizure Medications
Kerrie-Anne Ho, PhD, Patient Preference Lead at UCB, discusses preferences and perceptions of acute seizure medications. Data from interviews with people with seizures (PwS) and caregiver...
Pediatric Adrenal Insufficiency: Etiology, Diagnosis, and Management
Mitchell Geffner, MD, Co-Director, Congenital Adrenal Hyperplasia Clinic, and Ron Burkle Chair, Center for Endocrinology, Diabetes, and Metabolism, Children’s Hospital of Los Angeles, discusses the...
Efgartigimod in Juvenile Generalized Myasthenia Gravis
Abigail Schwaede, MD, Assistant Professor of Pediatrics at Northwestern University Feinberg School of Medicine, discusses results on intravenous efgartigimod in juvenile generalized myasthenia...
Maternal and Neonatal Outcomes of Chenodeoxycholic Acid Treatment in Pregnant Women With Cerebrotendinous Xanthomatosis
A study published in the Journal of Clinical Lipidology evaluated maternal and neonatal outcomes of chenodeoxycholic acid (CDCA) treatment during gestation in women with cerebrotendinous...
Prospective Cutaneous Lymphoma International Prognostic Index Study
Julia Scarisbrick, MD, MBhons, ChB, FRCP, is one of the leaders of the PROCLIPI (Prospective Cutaneous Lymphoma International Prognostic Index) study, a registry of patients with cutaneous T-cell...
Maralixbat’s Effect on Xanthoma Severity in Children With Alagille Syndrome
A study published in The Journal of Pediatrics describes a post hoc analysis of clinical trials evaluating maralixibat in children with Alagille syndrome and its effect on xanthoma severity....
FDA Approves Cavhanza (Nilotinib) Oral Tablet Formulation for First-Line Treatment of Ph+ Chronic Myeloid Leukemia
The US Food and Drug Administration (FDA) has approved Cavhanza (nilotinib) orally disintegrating tablets for the treatment of Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML)....
Results from the ADAPT SERON Clinical Trial of Efgartigimod IV in Patients With Seronegative Generalized Myasthenia Gravis
James Howard Jr., MD, Professor of Neurology at the University of North Carolina at Chapel Hill, discusses results from the ADAPT SERON clinical trial of efgartigimod IV in patients with...
METEOROID Clinical Trial of Enspryng (Satralizumab) in Patients With MOGAD
Michael Levy, MD, PhD, Neuroimmunologist and Associate Professor at Harvard Medical School, discusses results from the METEOROID clinical trial of Enspryng (satralizumab) in patients with myelin...
FDA Approves Pivekimab Sunirine for Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm
The US Food and Drug Administration (FDA) has approved pivekimab sunirine-pvzy for adults with blastic plasmacytoid dendritic cell neoplasm (BPDCN). BPDCN is a rare, aggressive CD4+ CD56+ myeloid...
New Trial to Compare Rituximab With Targeted Therapies in Patients With Neuromyelitis Optica Spectrum Disorder
Sumaira Ahmed, Founder and Executive Director of The Sumaira Foundation, discusses her organization and the BEST-NMOSD clinical trial for patients with neuromyelitis optica spectrum disorder...
Arginine Vasopressin Deficiency (AVP-D) Overview
Christopher Romero, MD, a pediatric endocrinologist at Mount Sinai Medical Center, New York City, and Associate Professor of Pediatrics at the Icahn School of Medicine at Mount Sinai discusses...
June Is CAH Awareness Month
June is CAH Awareness Month, a time dedicated to increasing understanding of congenital adrenal hyperplasia (CAH) and supporting the individuals and families impacted by this rare genetic condition....
DUET Clinical Trial of Xywav in Patients With Narcolepsy
Logan Schneider, MD, Adjunct Clinical Associate Professor of Psychiatry and Behavioral Sciences at Stanford University, discusses the DUET clinical trial of Xywav (low sodium oxybate) in patients...
Data Presented on Myasthenia Gravis at the 2026 American Academy of Neurology Meeting
Omar Sinno, MD, US Medical Strategy Lead for Rare Disease at UCB Pharma, discusses data presented on myasthenia gravis (MG) at the 2026 American Academy of Neurology meeting. MG is a...
Itvisma Gene Therapy for Patients With Spinal Muscular Atrophy
John Day, MD, PhD, Director of Neuromuscular Medicine at Stanford University, discusses the development of Itvisma gene therapy for patients with spinal muscular atrophy (SMA). SMA is...
Upcoming Regulatory Decision for LNTH-2501 in the Imaging of Neuroendocrine Tumors
Mauro Cives, MD, Associate Professor of Medical Oncology at the University of Bari, Italy, discusses the upcoming regulatory decision for LNTH-2501 to improve imaging of neuroendocrine tumors...
Results From the ACUITY Clinical Trial in Patients With Acute Optic Neuritis
Pablo Villoslada, MD, PhD, Founder and Medical Director of Accure Therapeutics and Head of Pathogenesis and New Therapies MS at IDIBAPS in Hospital Clínic in Barcelona, discusses results from the...
Outcomes of Pegcetacoplan Treatment in Adolescents With C3G and IC-MPGN
A recent publication in the Clinical Journal of the American Society of Nephrology presented outcomes of pegcetacoplan treatment in adolescents with C3 glomerulopathy (C3G) and primary immune...
Generalized Myasthenia Gravis: AANEM 2026 Highlights
CheckRare focused its AANEM 2026 meeting coverage on generalized myathesthenia gravis (gMG), and its treatment and management.
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TETON-2 Findings in Treatment of Idiopathic Pulmonary Fibrosis #podcast #raredisease
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Highlights from The Liver Meeting 2026 | The CheckRare Brief
Results of the Phase 3 OASIS Study of Oral Olorofim
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What should you know about myasthenia gravis?
From understanding the underlying disease mechanisms to exploring FDA-approved treatment options and ongoing clinical research, staying informed is essential for the rare disease community.
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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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