Quest Magazine

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:

ISIS-SOD1-Rx: So Far, So Good

Early results of a phase 1 clinical trial of ISIS-SOD1-Rx, an antisense oligonucleotide drug for SOD1-related amyotrophic lateral sclerosis (ALS), show the drug appears to be safe and well tolerated at the lowest dosage level, based on a group of six patients who received 12-hour infusions of it into their spinal fluid.

ALS TDI Webinar Discusses Current Research

The state of the science in ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease), current lines of research, efforts to establish a TDP43 research mouse colony, and a primer on protein-based therapies were the topics of a webcast on April 7, 2011, presented by ALS Therapy Development Institute (ALS TDI) CEO and Chief Scientific Officer Steve Perrin, and colleagues.

AAN Research Briefs on DM, DMD, LGMD, MG, MMD, SMA

Below are brief reports and links to more information about neuromuscular disease research presented at the 63rd annual meeting of the American Academy of Neurology (AAN), held in Honolulu April 9-16, 2011.

ALS Research Briefs from 2011 AAN Meeting

Neuralstem's trial of spinal cord injections of stem cells is on track

MDA Surveying Families about ‘Cost of Illness’

Most people affected by neuromuscular disease know all too well that their condition is costly. MDA wants to find out just how costly — and then translate those figures into terms that will speak to policy makers.

MDA Surveying Families about ‘Cost of Illness’

Most people affected by neuromuscular disease know all too well that their condition is costly. MDA wants to find out just how costly — and then translate those figures into terms that will speak to policy makers.

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