Quest Magazine

'Antisense' Ameliorates SMA Symptoms in Mice

A team of research scientists has found that mice with a disease resembling a severe form of spinal muscular atrophy (SMA) that were treated with a gene-modifying molecule produced more of a needed protein throughout their spinal cords; developed bigger, stronger muscles; and survived longer than expected. 

IL10 Protein May Influence DMD Severity

A team of researchers at the David Geffen School of Medicine at the University of California-Los Angeles (UCLA) has demonstrated that the naturally occurring protein interleukin 10 (IL10) may help reduce harmful inflammation and promote muscle regeneration in people with Duchenne muscular dystrophy (DMD), and potentially those with other forms of muscular dystrophy.

MDA supported James Tidball, director of the Duchenne Muscular Dystrophy Research Center at UCLA, for this work.

Boy with DMD 'Outswims' Olympian Michael Phelps

Logan Mitzel has one word for his swim with Olympic champion Michael Phelps: “awesome.”

The 11-year-old from Aurora, Ill., who has Duchenne muscular dystrophy (DMD) and relies on a manual wheelchair for mobility, loves to swim so much that in 2010 he completed a streak of swimming every day for 77 consecutive days.

Reactivated Virus May Contribute to ALS

New research suggests that the DNA of a normally dormant virus may be playing a role in causing amyotrophic lateral sclerosis (ALS).

If the findings are confirmed, they could ultimately have implications for treatment of the disease.

About the HERV-K virus

'Cloud-Enhanced' Accessibility: Competition Seeks Visionaries

The Federal Communications Commission wants to know: What’s your vision for using “cloud computing” to create new opportunities for people with disabilities?

The FCC is co-sponsoring the competition “Lifted by the Cloud: Visions of Cloud-Enhanced Accessibility,” and asking participants to create short multimedia presentations showing “what could be possible using the computing and communication power now available via the Internet.”

ALS Research Briefs

Loss of TDP43's normal function could be ALS contributor

Legislative Briefs: International Rare Disease Day and More

International Rare Disease Day 2011

Monday, February 28, 2011, marks the fourth annual International Rare Disease Day, in which hundreds of patient organizations from more than 40 countries conduct awareness-raising activities around the slogan “Rare but Equal.” The worldwide event is an effort to draw attention to rare diseases and the millions of people who are affected by them — including all those covered by the MDA umbrella. 

Research Briefs: CMS, DMD, LGMD, Pompe, Stem Cells

Congenital myasthenic syndromes

A multinational team of scientists has identified mutations in the gene for glutamine-fructose-6-phosphate transaminase 1 (GFPT1) as responsible for some forms of a congenital myasthenic syndrome (CMS).

Unconventional Pianist with CMT Hopes to Raise a Million

Amble playing a "techno music" piece that he composed when he was 9.

Research Briefs: CMT, CMS, DMD/BMD, FA, Pompe disease, SBMA

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