Duchenne Muscular Dystrophy (DMD)

Closer Than Ever Before

PTC Announces Phase 3 Trial Results for Ataluren in DMD

BioMarin, Sarepta Announce Dates for FDA Advisory Committee Meetings

Sarepta Reports Data from Phase 2 Trial of Eteplirsen for Treatment of DMD

$2 Million in New MDA Grants Target DMD

FDA Accepts Sarepta NDA for Eteplirsen to Treat DMD

Background: Sarepta announced today that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for eteplirsen to treat Duchenne muscular dystrophy (DMD).

Eteplirsen is an “exon-skipping” drug that targets a section of DNA called exon 51, and may help up to 13 percent of Duchenne muscular dystrophy (DMD) patients.

DMD - Dada Pisconti, Ph.D.

Dada Pisconti, at the University of Liverpool, United Kingdom, was awarded an MDA research grant totaling $300,000 over three years to study how the environment established by fibrosis and inflammation affects the maintenance and regenerative properties of muscle stem cells in Duchenne muscular dystrophy (DMD). Pisconti will work to determine whether serine protease inhibitor proteins are involved in the DMD disease process.

Marathon Pharmaceuticals Begins New Drug Application Process for Deflazacort to Treat Duchenne Muscular Dystrophy

Following a pre-NDA meeting with the U.S. Food and Drug Administration (FDA), Marathon Pharmaceuticals has announced that it will begin the New Drug Application (NDA) process for deflazacort as a treatment for Duchenne muscular dystrophy (DMD).

Marathon expects to submit the NDA in the first quarter of 2016. If the application is approved, the company could make the drug commercially available in the United States in the first quarter of 2017.

DMD - James Ervasti, Ph.D.

James Ervasti, professor in the department of biochemistry, molecular biology & biophysics at the University of Minnesota in Minneapolis, was awarded an MDA research grant totaling $300,000 over three years to develop methods of identifying non-invasive biomarkers that can be measured in all patients across the entire spectrum of Duchenne muscular dystrophy (DMD) disease severity.

DMD - Natassia Vieira, Ph.D.

Natassia Vieira, an investigator at Biosciences Institute, University of São Paulo - São Paulo, Brazil, was awarded an MDA development grant totaling $180,000 to study a genetic mechanism that appears to compensate for mutations in the gene that causes Duchenne muscular dystrophy (DMD). Vieira will study the mechanism by which overexpression of a protein called Jagged1 protects dogs and fish carrying a DMD-causing mutation from exhibiting the typical symptoms and progression of the disease.