Strongly


An expression of the live unlimited spirit, stories and voices of the MDA community

Five Questions with Researcher Criss Hartzell

Criss Hartzell, professor of cell biology at Emory University School of Medicine in Atlanta, Ga., recently was awarded an MDA research grant to study the ANO5 gene. Mutations in the gene have been implicated in both type 2L limb-girdle muscular dystrophy (LGMD) and a subtype of Miyoshi distal muscular dystrophy (a type of distal muscular dystrophy, . . .

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BioMarin Receives Favorable Ruling in the Use of Exon 51 Antisense Oligonucleotides Patent Interference

Priority of U.S. Patent Claims Confirmed by U.S. Patent Trial and Appeal Board (PTAB) SAN RAFAEL, Calif., Sept. 29, 2015 (GLOBE NEWSWIRE) — BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the Patent Trial and Appeal Board (PTAB) issued a decision in favor of BioMarin’s claims to the use of exon 51 antisense oligonucleotides to treat . . .

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

Alliance is part of an MDA initiative to establish new collaborative partnerships CHICAGO, September 2, 2015 – The Muscular Dystrophy Association (MDA) and the Friedreich’s Ataxia Research Alliance (FARA) today announced a partnership that will pave the way for advancements in Friedreich’s Ataxia (FA) research, therapeutic development and clinical care, thus deepening understanding of the . . .

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$2 Million in New MDA Grants Target DMD

By Laura Hagerty, Ph.D., MDA’s DMD Scientific Program Officer On September 7, 2015, MDA will rally alongside our Duchenne muscular dystrophy (DMD) community — kids, adults, families, caregivers and others — as we recognize World Duchenne Awareness Day. And as we move toward that day, there’s a palpable feeling of excitement in the air. For . . .

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MDA Appoints Leaders From Across the Country to Board of Directors

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 . . .

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MDA Awards $10 million in new Research Grants

Record-breaking interest highlights progress, desire to find breakthroughs across diseases CHICAGO, August 25, 2015 – Powered by its big-picture perspective to accelerate treatments and cures across the broad spectrum of neuromuscular diseases, MDA today announced the award of $10 million in new research grants to the world’s brightest scientists conducting leading-edge discovery for muscular dystrophy, ALS and . . .

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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. Exon . . .

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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 . . .

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