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

BioBlast Is Developing an OPMD Drug

BioBlast Is Developing an OPMD Drug

Israeli biotechnology company BioBlast is developing and testing Cabaletta, designed to counteract abnormal clumping of cellular proteins in oculopharnyngeal muscular dystrophy (OPMD)

AveXis SMA Gene Therapy Trial Continues Recruiting

AveXis SMA Gene Therapy Trial Continues Recruiting

The first three infants in a gene therapy trial for spinal muscular atrophy have been treated; the trial continues, and additional trials are planned

Drug Development for DMD: Fall 2014 Update

Drug Development for DMD: Fall 2014 Update

Compounds designed to alter genetic instructions for dystrophin, increase production of utrophin, or prevent scar tissue formation in muscle are moving through the development pipeline

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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