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

Trial of Antisense Drug for Type 1 Myotonic Dystrophy Opens

Trial of Antisense Drug for Type 1 Myotonic Dystrophy Opens

Isis Pharmaceuticals has announced the opening of a U.S. trial to evaluate the safety and tolerability of ISIS-DMPKRx in adults with type 1 myotonic dystrophy

Myostatin Blocker To Be Tested in DMD

Myostatin Blocker To Be Tested in DMD

Pfizer will test its experimental drug PF-06252616 in ambulatory boys with Duchenne MD at multiple sites in the U.S. and elsewhere

DMD: Utrophin Modulator SMT C1100 To Undergo Further Testing

DMD: Utrophin Modulator SMT C1100 To Undergo Further Testing

MDA-supported Summit PLC announced it will continue testing it experimental drug in Duchenne MD patients, along with dietary modifications that may improve drug absorption

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