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

Drug Development for DMD: February 2015 Update

Drug Development for DMD: February 2015 Update

This report on treatment development for Duchenne muscular dystrophy contains news about fighting fibrosis (scarring), treating DMD-related heart disease, exon skipping and the corticosteroid deflazacort

DMD: Eteplirsen Data Still Encouraging After Three Years

DMD: Eteplirsen Data Still Encouraging After Three Years

Boys with DMD continuously treated with eteplirsen for three years walked farther in six minutes than those receiving a placebo for the first six months

Stanford Biobank Seeks Tissue Samples

Stanford Biobank Seeks Tissue Samples

Stanford University is collecting samples of blood, muscle and other tissues from people with neuromuscular disorders for use in research

PTC Begins Submission of New Drug Application for DMD Stop Codon Read-Through Drug

PTC Begins Submission of New Drug Application for DMD Stop Codon Read-Through Drug

PTC Therapeutics has begun submitting an application for U.S. approval of Translarna (ataluren) for Duchenne MD; completion of the application is slated for late 2015

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