AMO Pharma has announced the initiation of REACH-CDM, a pivotal Phase 2/3 clinical trial that will assess the efficacy…
Articles by Marisa Wexler, MS
Translarna (ataluren) has been approved in Russia for treating Duchenne muscular dystrophy (DMD) caused by nonsense mutations in patients…
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops…
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like…
In a Facebook Live event hosted by the Muscular Dystrophy Association (MDA), experts discussed the effect that the…
Real-world and natural history data could be used to supplement, or perhaps replace, the use of a placebo in future…
An imbalance in cells producing proteins called aldehyde dehydrogenases (ALDHs) — favoring fat formation in tissue — is evident…
An application requesting the approval of casimersen (SRP-4045) to treat Duchenne muscular dystrophy (DMD) amenable to exon 45 skipping has…
The LELANTOS Phase 3 clinical trial, which is evaluating FibroGen‘s pamrevlumab in people with Duchenne muscular dystrophy…
The importance of communication and self-care for people with muscular dystrophy and other neuromuscular diseases who are pursuing…