Quest Magazine

MDA Awards Grant to Test MTM Gene Transfer in Dogs

MDA’s translational research program has announced it is funding research into a potential treatment for the inherited muscle disorder X-linked myotubular myopathy (MTM).

The grant of $369,365 grant to Wake Forest University professor Martin Childers will fund a three-year study of myotubularin gene transfer in mice and dogs that have an MTM-like disease.

Friends with SMA Inspire Scouts to Create Wheelchair Device

Ten-year-old Quinn Schnitzlein has been using a power chair since he was 3, so he’s a pretty good driver. But the boy from Marietta, Ga., who has spinal muscular atrophy (SMA), often bumped into things when he backed up.

One day last year, Quinn and some friends were at a fast-food restaurant. “We were at different tables,” Quinn says, “and when I’d try to turn around to talk to them, I kept hitting things.”

May 25 Webinar Focuses on Newly Diagnosed

Every day in the United States, about 13 people receive a diagnosis of ALS (amyotrophic lateral sclerosis, or Lou Gehrig’s disease).

That’s 5,000 Americans a year who are new to this disease. MDA's ALS Division wants to reach out to these people and their families, encourage and support them, and most importantly, let them know they’re not alone.

FDA Approves Phase 1 Clinical Trial of RG3039 in SMA

In a historic first, biotech company Repligen Corp., of Waltham, Mass., has received approval from the U.S. Food and Drug Administration (FDA) to begin a phase 1 clinical trial of the experimental drug RG3039 for spinal muscular atrophy (SMA).

MDA Presents Housing Rights Webinar May 24

Are owners of apartment buildings required to have accessible units? Can they charge higher rent for them? And can they refuse to rent to a person with a service dog?

These are just some of many questions about housing rights that will be answered Tuesday, May 24, 2011, during an MDA-sponsored webinar titled "Know Your Rights — The Fair Housing Act," beginning at 4 p.m. EDT, 2 p.m. MDT and 1 p.m. PDT.

ALS Research Briefs

NurOwn stem cell therapy trial set to begin

Biotechnology company Brainstorm Cell Therapeutics announced May 17, 2011, that it has received approval from Israel's Ministry of Health to conduct a phase 1-2 clinical trial of stem cell therapy in adults with ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease).

MDA Launches Transitions Resource Center for Young Adults

Navigating the transition from childhood to adulthood can be complicated. Young people confront numerous important changes at this time, including going off to college, moving away from home, starting a career, establishing friendships and falling in love.

Young adults with disabilities may find it particularly challenging to find and utilize all available resources to help manage and coordinate these changes.

Research Briefs: BMD, DMD, EDMD, FA, LGMD, OPMD, Pompe disease, SMA

Idebenone may help maintain respiratory function in DMD

Santhera Pharmaceuticals announced May 9, 2011, that its drug Catena (generic name idebenone) appears to slow the decline in respiratory function associated with aging in people with Duchenne muscular dystrophy (DMD). Idebenone may improve energy production in muscle and nerve cells.

FUS and ALS: What's the Connection?

 The gene for FUS  was associated in 2009 with some forms of ALS (amyotrophic lateral sclerosis, or Lou Gehrig’s disease).

Now, overlapping findings from four recent studies have revealed tantalizing clues about the molecular underpinnings of FUS-related forms of the disease.

ALS Research Briefs

Patients Like Me online lithium study utilized social media

An observational study of data self-reported on the online forum Patients Like Me by people with ALS who elected to take the drug lithium carbonate has shown two things: