September is Muscular Dystrophy Awareness Month, and advocates are launching campaigns to educate people about the different types of rare…
Articles by Marisa Horak, MS
For the first time, a person with Duchenne muscular dystrophy (DMD) has been given an experimental gene-editing therapy, called…
The U.S. Food and Drug Administration (FDA) is extending its review of deramiocel, an experimental cell therapy for…
Epicrispr Biotechnologies has raised $90 million in financing to support late-stage clinical testing of EPI-321, the company’s epigenetic treatment…
A U.S. biopharmaceutical company is gearing up to launch its first clinical trial of a novel cell therapy designed to…
The University of Alabama at Birmingham (UAB) has launched an expanded access program to make the experimental exon-skipping therapy…
An advisory committee to the U.S. Food and Drug Administration (FDA) has voted that available evidence does not support the…
The next Clinical & Scientific Conference hosted by the Muscular Dystrophy Association (MDA) will take place March 21-24, 2027,…
Continuing corticosteroid treatment into adulthood was associated with slower lung-function decline in people with Duchenne…
The U.S. Food and Drug Administration (FDA) is considering whether to conditionally approve zeleciment rostudirsen (z-rostudirsen), an experimental therapy for…