Treatment with Translarna (ataluren) increased levels of the protein dystrophin in young boys with Duchenne muscular dystrophy (DMD)…
Articles by Marisa Wexler, MS
Applications are now open for the seventh annual Strategies to Realize Innovation, Vision, and Empowerment (STRIVE) Awards program for…
A potential exon-skipping therapy for Duchenne muscular dystrophy (DMD) called DS-5141 showed a good safety profile with repeat dosing in a…
AMO Pharma has announced the initiation of REACH-CDM, a pivotal Phase 2/3 clinical trial that will assess the efficacy…
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…