by Peter Ciszewski | May 6, 2021
Pritesh J. Gandhi, PharmD, Former Vice President and General Manager, Lumasiran Program at Alnylam Pharmaceuticals, discusses the results of the Illuminate-A study, a Phase 3 randomized, double-blind, placebo-controlled study that evaluated the efficacy and...
by Peter Ciszewski | May 5, 2021
Matthew Klein, MD, Chief Development Officer at PTC Therapeutics, explains the mechanism of action of risdiplam (Evrysdi), an approved daily therapy for spinal muscular atrophy (SMA) in patients 2 months and older. SMA is a rare inherited neuromuscular disorder...
by Peter Ciszewski | May 5, 2021
Michael Hayden, MBChB, PhD, CEO of Prilenia Therapeutics and a Killam Professor at the University of British Columbia, describes the current research landscape for Huntington disease. Huntington disease is an inherited, neurodegenerative disorder that usually...
by Peter Ciszewski | May 4, 2021
Sue Apkon, MD, Chief of Pediatric Rehabilitation Medicine, Children’s Hospital Colorado, discusses a real-world study of patients with Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) who switched from prednisone to deflazacort. The data...
by Peter Ciszewski | May 3, 2021
Howard Trachtman, MD, from NYU Langone Health, provides an overview of the safety and efficacy of sparsentan to treat focal segmental glomerulosclerosis (FSGS). FSGS is a heterogeneous kidney disorder that often presents with a variable degree of proteinuria...