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

FEATURED IN QUEST MAGAZINE

Power wheelchair options and benefits for toddlers

posted on January 5, 2015 - 9:12am
Note: The products mentioned in this article are not endorsed by MDA. When choosing any assistive technology equipment, be sure to do your research and consult with your MDA clinic team, as well as an assistive technology professional (ATP).

For one mother living with Charcot-Marie-Tooth disease, daily perseverance starts with redefining 'normal'

posted on January 5, 2015 - 9:11am
I measure and compare myself to other “normal” mothers out there — those who are able-bodied. It’s hard not to, especially when it comes to doing everyday things with my son.

Meet Grace Pavlath, MDA's new scientific director

posted on January 5, 2015 - 9:09am
Grace Pavlath, an accomplished muscle disease researcher and a professor in the Department of Pharmacology at Emory University School of Medicine in Atlanta, recently joined MDA as senior vice president and scientific program director. She will be based in Atlanta initially and will eventually relocate to Washington, D.C.

Research updates and clinical trials information

posted on January 5, 2015 - 9:07am
What About My Disease? Readers sometimes wonder what’s happening with research for their diseases when they don’t see news about them for a while in the pages of Quest. But keep in mind: Research that seems to be for one disorder often has spillover implications for others.

News and updates from the MDA community

posted on January 5, 2015 - 9:05am
Shining STAR: The MD STARnet project expands to learn more about neuromuscular disease

Feedback from Quest readers and the MDA community

posted on January 5, 2015 - 9:03am
Letters to the Editor: Hitting home