Boys and young men with Duchenne muscular dystrophy (DMD) who switched from a placebo to the experimental cell therapy…
Articles by Marisa Horak, MS
The U.S. Food and Drug Administration (FDA) has given the go-ahead for a mid-stage clinical trial testing the oral therapy…
Twenty college students with Duchenne muscular dystrophy (DMD) will receive awards of up to $5,000 through Sarepta Therapeutics‘…
A Phase 3 clinical trial testing delpacibart etedesiran (del-desiran), an experimental treatment for myotonic dystrophy type 1 (DM1), failed…
The U.S. Food and Drug Administration (FDA) has granted both fast track and orphan drug designations to Scholar Rock‘s…
September is Muscular Dystrophy Awareness Month, and advocates are launching campaigns to educate people about the different types of rare…
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…