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

Drug Development for DMD: Fall 2014 Update-Part 2

Drug Development for DMD: Fall 2014 Update-Part 2

In this second of two fall reports on treatment development for Duchenne muscular dystrophy, news about ARM210, CAP-1002, PDE5 inhibitors, drisapersen and CAT-1004 is presented

Sarepta Reaffirms Commitment to Eteplirsen, Other DMD Drugs

Sarepta Reaffirms Commitment to Eteplirsen, Other DMD Drugs

In a Dec. 2, 2014, webcast for the Duchenne MD community, Sarepta Therapeutics explains plans, answers questions; the presentation is available on Sarepta's site

Phase 3 Trial of ISIS-SMNRx Now Open for Children with SMA

Phase 3 Trial of ISIS-SMNRx Now Open for Children with SMA

Isis Pharmaceuticals has opened a second phase 3 trial to test its "antisense" drug for spinal muscular atrophy in children ages 2 to 12

PTC To Test RG7800 in SMA

PTC To Test RG7800 in SMA

The investigational compound is designed to raise levels of the SMN protein, a lack of which is the underlying cause of spinal muscular atrophy (SMA)

BMD: Follistatin Gene Transfer Results Encouraging

BMD: Follistatin Gene Transfer Results Encouraging

Four of six men with Becker muscular dystrophy increased their six-minute walking distance after injections of follistatin genes into the thigh muscles of both legs

FEATURED IN QUEST MAGAZINE

Websites for mapping and researching accessible places

posted on October 30, 2014 - 9:07am
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 with an assistive technology professional (ATP). Between fall road trips and upcoming holiday travel, wouldn’t it be great to know the accessibility features of every place you might go before...

News, events and updates from the MDA community

posted on October 30, 2014 - 9:05am
Hustle for Muscle: A family affected by MD forms endurance running team to raise money for MDA

Feedback from Quest readers and the MDA community

posted on October 30, 2014 - 9:03am
Letters to the Editor: Adventures in parenthood Editor’s note: In response to the summer issue of Quest’s cover story, A Dream Within Reach, about the challenges and rewards of becoming a parent when you have a neuromuscular disease, we received letters and comments online at quest.mda.org that were overwhelmingly supportive. Here are two responses: 

Research updates and clinical trials information

posted on October 30, 2014 - 9:01am
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.

Challenge Accepted: Finding inspiration and hope in the ALS Ice Bucket Challenge

posted on October 30, 2014 - 9:00am