Quest Magazine

$2 Million in New MDA Grants Target DMD

MDA and FARA Partner to Advance Treatments and Care for Friedreich’s Ataxia

MDA Targets $2 Million in New Grants Toward Understanding and Treating ALS

MDA Appoints Leaders From Across the Country to Board of Directors

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.

MDA Awards $10 million in new Research Grants

CHICAGO, August, 27, 2015 — Prominent business and community leaders from across the country have been appointed to the Muscular Dystrophy Association’s Board of Directors. As members of the board, these 20 men and women will volunteer assistance and counsel to support MDA’s mission to fund lifesaving research, provide comprehensive care through its services and programs, and foster independence within its community.

Strength in Numbers: New Grants Pave the Way Toward Treatments and Cures

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.

FDA Approves Drug for Primary Hyperkalemic and Hypokalemic Periodic Paralysis

Statement from MDA Executive Vice President and Chief Medical and Scientific Officer Valerie A. Cwik, M.D.:

We couldn’t be more pleased for our families affected by hyperkalemic and hypokalemic periodic paralysis, as the first treatment for these diseases has been approved by the U.S. Food and Drug Administration (FDA) and is expected to be available in the coming months."

Becker Muscular Dystrophy Research Today