Dyne Therapeutics‘ experimental therapy zeleciment basivarsen (z-basivarsen) may help improve multiple measures of physical function in people with…
Articles by Marisa Horak, MS
Mental health problems such as anxiety and depression are common for people with muscular dystrophy, affecting most individuals in…
The gene therapy Elevidys (delandistrogene moxeparvovec-rokl), approved in the U.S. to treat people ages 4 and older with…
Boys and young men with Duchenne muscular dystrophy (DMD) who switched from a placebo to the experimental cell therapy…
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…