New Guideline for CMD Diagnosis and Care

New Guideline for CMD Diagnosis and Care

A committee convened by the American Academy of Neurology has published new recommendations for doctors who see children with congenital muscular dystrophy

CMD: Phase 1 Omigapil Trial Remains Open

CMD: Phase 1 Omigapil Trial Remains Open

Santhera Pharmaceuticals is testing anti-cell death compound omigapil in children and teens with merosin-deficient and Ullrich congenital muscular dystrophies

FDA OKs Testing of OPMD Drug in US

FDA OKs Testing of OPMD Drug in US

Israel-based BioBlast Pharma says a California site will be added soon to locations already testing Cabaletta in oculopharyngeal muscular dystrophy in Israel and Canada

SMA: ISIS-SMNRx To Be Tested in Presymptomatic Infants

SMA: ISIS-SMNRx To Be Tested in Presymptomatic Infants

The experimental antisense drug will be tested in infants with a genetic spinal muscular atrophy diagnosis who are to date without symptoms

DMD: New Strategy Aims to Change Dystrophin DNA

DMD: New Strategy Aims to Change Dystrophin DNA

MDA-supported researcher Charles Gersbach and colleagues have developed a strategy to change dystrophin DNA that has shown promise in the lab

NIH Will Test Antioxidant Drug in Central Core Disease

NIH Will Test Antioxidant Drug in Central Core Disease

A drug that has FDA approval for other indications is being tested in CCD patients who are at least 7 years old

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Celebrating scientific advancement, MDA summer camp and more

posted on April 21, 2015 - 9:00am
I am often asked what’s the single most important way we at MDA can best serve our families and best represent our donors. While MDA is delighted to provide an array of services and support, we believe our greatest opportunity for impact is to mobilize aggressively on the front lines in the discovery of treatments and cures.