Duchenne Muscular Dystrophy (DMD)

Sarepta Completes NDA for Eteplirsen and FDA Accepts BioMarin NDA for Drisapersen; Two DMD Treatments on Horizon

Background: On Monday, June 29, BioMarin announced  that the FDA has accepted their New Drug Application (NDA) for drisapersen and Sarepta announced that they have completed their NDA submission for eteplirsen. Both "exon-skipping" drugs target exon 51, and may help up to 13% of Duchenne muscular dystrophy patients.

Akashi Therapeutics Reports Data from Phase 1b-2a Study in Boys with Duchenne Muscular Dystrophy

BioMarin Completes Rolling NDA Submission for Treatment of Duchenne Muscular Dystrophy

Santhera Reports Positive Phase III Clinical Trial Results for Raxone®/Cantana® (idebenone) for the Treatment of Duchenne Muscular Dystrophy

Santhera Receives FDA Fast Track Designation for Treatment of Duchenne Muscular Dystrophy

DMD: New Strategy Aims to Change Dystrophin DNA

MDA research grantee Charles Gersbach, assistant professor of biomedical engineering at Duke University, and colleagues, recently announced an advance in gene modification that could turn out to be a game-changer for boys and young men with Duchenne muscular dystrophy (DMD). The team's results were published Feb.

ReveraGen BioPharma Announces Start of Phase 1 Clinical Trial of VBP15 Dissociative Steroid Drug

Drug Development for DMD: February 2015 Update

Development of treatments for Duchenne muscular dystrophy (DMD) continues to advance. Many of the new investigational drugs are potentially applicable to all DMD patients, while a few target those with specific mutations in the dystrophin gene.

Fighting fibrosis

DMD: Eteplirsen Data Still Encouraging After Three Years

Boys with Duchenne muscular dystrophy (DMD) caused by specific genetic mutations who were treated with the experimental drug eteplirsen for 168 weeks (3.2 years) continued to show an advantage in the distance they were able to walk compared to those treated with the drug for only 144 weeks (2.8 years), although all boys had declined in walking distance since the start of this phase 2b trial of eteplirsen.

PTC Begins Submission of New Drug Application for DMD Stop Codon Read-Through Drug

New Jersey-based PTC Therapeutics has begun submitting a rolling new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of its experimental drug for the treatment of Duchenne muscular dystrophy (DMD) caused by specific flaws (mutations) in the dystrophin gene.

Pages