News

Sarepta stops development of exon 51-skipping therapy for DMD

Despite positive trial data, Sarepta Therapeutics has decided to stop the clinical development of SRP-5051 (vesleteplirsen), an exon 51-skipping therapy for some people with Duchenne muscular dystrophy (DMD). While increases in dystrophin levels seen with the exon-skipping treatment were encouraging, according to the company, concerns over the…

CureDuchenne partners on World of Warcraft research fundraiser

CureDuchenne is partnering with Blizzard Entertainment, maker of the massively multiplayer online role-playing game World of Warcraft, on a fundraiser to help advance research into new treatments for Duchenne muscular dystrophy (DMD). Through Jan. 7, players of the video game will have the opportunity to adopt an in-game pet…

FDA awards LAMA2-CMD therapy MDL-101 orphan drug status

MDL-101, Modalis Therapeutics’ epigenetic editing therapy for LAMA2-related congenital muscular dystrophy (LAMA2-CMD), has been granted orphan drug status by the U.S. Food and Drug Administration (FDA). The designation is intended to encourage the development of therapies for serious or life-threatening rare diseases, which are those affecting fewer than…

EMA agency continues to oppose renewing Translarna’s approval

The Committee for Medicinal Products for Human Use (CHMP) — part of the European Medicines Agency — again maintained its recommendation not to renew conditional marketing authorization for Translarna (ataluren), a treatment for Duchenne muscular dystrophy (DMD) caused by nonsense mutations. The European Commission, which has final say…

RNA-based approach shows promise for DM1 in cell models

Treatment with antimiRs, an RNA-based therapy approach, was able to correct molecular defects in a cell model of myotonic dystrophy type 1 (DM1) — a form of muscular dystrophy — according to the results of a new study. The researchers called their work “a significant step forward,” and noted that…