Current Status of Gene Therapies in Rare Neuromuscular Disorders ++++

FEATURED ARTICLES Current Status of Gene Therapies in Rare Neuromuscular Disorders https://checkrare.com/current-status-of-gene-therapy-in-rare-neuromuscular-disorders/ Alan Beggs, PhD and Julie A Parsons, MD, discuss the current status of gene therapies in rare neuromuscular disorders in this eight-part podcast series. Current Status of Gene Therapy in Lysosomal Storage Disorders https://checkrare.com/current-status-of-gene-therapy-in-lysosomal-storage-disorders/ Nicola Longo MD, PhD and Mark Roberts, MD discuss the current status of gene therapies in lysosomal storage disorders. Catching the Clues, Changing the Course of Lysosomal Storage Disorders https://checkrare.com/catching-the-clues-changing-the-course-of-lysosomal-storage-disorders/ Yoshikatsu Eto, MD, PhD, Nicole Muschol, MD, Patrício Aguiar, MD, and Robert Hopkin, MD, explore the patient journey across the lysosomal storage disorder (LSD) continuum, focusing on persistent gaps in recognition, diagnosis, timely treatment initiation, and long-term care. Consider Rare: Suspecting and Diagnosing Fibrodysplasia Ossificans Progressiva (FOP) https://checkrare.com/suspecting-and-diagnosing-fibrodysplasia-ossificans-progressiva-fop/ Ellen Elias, MD, Professor, Pediatrics and Genetics, University of Colorado School of Medicine, and Christiaan Scott, MD, Professor of Medicine at the University of Ottawa, examine best practices to suspect and diagnose this ultra-rare condition.