Alaa Hamed, Global Head of Medical Affairs for Rare Diseases for Sanofi, discusses two clinical trials that reinforce positive data for hemophilia treatments. #CheckRare #RareDisease #Hemophilia checkrare.com/clinical-trial…

Stephen Russell, MD, Carver College of Medicine, discusses long-term data on Luxturna (voretigene neparvovec-rzyl) for retinal dystrophy treatment. #CheckRare #RareDisease #RetinalDystrophy checkrare.com/gene-therapy-l…

Dana Rizk, MD, Professor of Medicine at UAB Medicine, discusses the U.S. Food and Drug Administration (FDA) accelerated approval of Fabhalta (iptacopan) for the reduction of proteinuria in adults with IgA nephropathy. #IgAN #CheckRare #RareDisease checkrare.com/accelerated-ap…

CME Program: Gustavo Maegawa, MD, Andrea Dardis, PhD, and Mia Horowitz, PhD discuss Biomarkers and Other Genetic Approaches in LDs #CheckRare #RareDisease #CME checkrare.com/learning/p-gri…

Corey Cutler, MD, Director of Adult Stem Cell Transplantation Program at Dana-Farber and a Professor of Medicine at Harvard Medical School, discusses the approval of Niktimvo (axatilimab) for the treatment of chronic graft versus host disease. #GVHD #CheckRare checkrare.com/fda-approves-t…

Dean Suhr, Co-founder and President of the MLD Foundation, highlights how the cost struggle associated with expensive therapies, such as the recently approved gene therapy, Lenmeldy (atidarsagene autotemcel), to treat metachromatic leukodystrophy. checkrare.com/gene-therapy-f…

Ponni Subbiah, MD, Senior Vice President of Acadia Pharmaceuticals, Global Head of Medical Affairs and Chief Medical Officer, discusses long-term data from clinical trial extension of trofinetide in patients with Rett syndrome. #CheckRare #RareDisease #RettSyndrome checkrare.com/long-term-data…

Diane Schriner, Vice President of Erdheim-Chester Disease Global Alliance, provides an overview of Erdheim-Chester disease #ECD #CheckRare #RareDisease checkrare.com/overview-of-er…

Jula Inrig, MD, Chief Medical Officer at Travere Therapeutics, discusses a clinical trial that tested sparsentan for treatment of IgA nephropathy, compared to traditional RAS inhibitors. #CheckRare #RareDisease #IgANephropathy checkrare.com/clinical-trial…

Diego Ferone, MD, PhD, University of Genoa and Chief of the Endocrine Unit at San Martino Hospital, discusses a novel octreotide formulation for the treatment of acromegaly. #CheckRare #RareDisease #Acromegaly checkrare.com/clinical-trial…

Hideki Garren, MD, PhD, Chief Medical Officer for Prothena, discusses the pathophysiology of AL amyloidosis and the mechanism of action of investigational drug birtamimab. #CheckRare #RareDisease #ALAmyloidosis checkrare.com/pathophysiolog…

Pisit “Duke” Pitukcheewanont, MD, Chief Medical Officer for Lumos Pharma, discusses a phase 2 trial evaluating LUM-201 (ibutamoren) for treatment of growth hormone deficiency. #CheckRare #RareDisease #GHD checkrare.com/phase-2-trial-…

Hypoparathyroidism is a rare endocrine disorder characterized by a deficiency of parathyroid hormone, which leads to decreased serum calcium and increased phosphorus levels in the blood. #CheckRare #RareDisease #Hypoparathyroidism checkrare.com/hypoparathyroi…

Ben Zimmer, CEO of Priovant Therapeutics, discusses the VALOR clinical trial testing investigational therapy for dermatomyositis treatment, brepocitinib. #CheckRare #RareDisease #Dermatomyositis checkrare.com/investigationa…

Prem Subramanian, MD, PhD, Professor of Ophthalmology at CU School of Medicine, discusses long-term data testing teprotumumab in patients with thyroid eye disease. #TED #CheckRare #RareDisease checkrare.com/long-term-data…

Richard Wasserman, MD, PhD, Medical Director of Pediatric Allergy and Immunology at Medical City Children’s Hospital, discusses the FDA expanded label approval for Xembify for primary immunodeficiencies #CheckRare #RareDisease #PIDs checkrare.com/expanded-label…

Frank Rivera, Founder of Sarcoidosis of Long Island and Stronger Than Sarcoidosis and patient advocate, discusses how his organization fulfills the unmet needs of sarcoidosis patients. #CheckRare #RareDisease #Sarcoidosis checkrare.com/fulfilling-the…

Hana Weltin, Intern at Beyond the Diagnosis, discusses how she advocates for herself and others in the rare disease space. #CheckRare #RareDisease #EhlersDanlos checkrare.com/being-an-advoc…

Mario Maldonado, MD, Head of Clinical Development for Global Endocrinology at Recordati Rare Diseases, discusses current treatment options for patients with Cushing’s syndrome. #CheckRare #RareDisease #CushingsSyndrome checkrare.com/current-treatm…

Leslie Edwin, President of the Cushing’s Support and Research Foundation (CSRF), and Gretchen Jordan, Associate Director of the CSRF, discuss their personal diagnostic journeys. #CheckRare #RareDisease #Cushings checkrare.com/the-diagnostic…