Iowa City, IA-The development of gene-directed therapy for common genetic ocular diseases that will be deployable on a population scale rests on coordinated research involving a series of steps. However, judging from progress made so far, such modalities might become clinically available soon, said Edwin M. Stone, MD, PhD.
Exploring New Pathways in Muscular Dystrophy Research: From Fibrosis to Function
April 30th 2025Matthew Alexander, PhD, a neuromuscular expert, discussed the evolving landscape of targeted therapies in muscular dystrophies, from fibrosis and glycosylation to combination strategies with gene therapy.