Quest Magazine

MDA/ALS Research Center Earns Prestigious Award

The Forbes Norris MDA/ALS Research and Treatment Center in San Francisco has received Program of the Year honors from the California Speech-Language-Hearing Association (CSHA).

MDA Labor Day Telethon Moves to Shorter Format

Sometimes, less is more — and that’s the strategy behind MDA’s decision to shave off more than 15 hours from next year’s Labor Day Telethon broadcast.

The new six-hour Telethon will run during the prime-time viewing hours of 6 p.m. to midnight (Eastern time) on the Sunday of Labor Day weekend, and will include the same time-tested mix of top entertainment, profiles of individuals and families with neuromuscular diseases, check presentations by MDA sponsors and live local segments.

Immune Response Must Be Considered in DMD Gene Therapy

Editor's note: This story was updated April 27, 2012.

Immune-system rejection of newly synthesized dystrophin protein occurred in at least some of the boys with Duchenne muscular dystrophy (DMD) who participated in a safety trial of dystrophin gene therapy.

The finding was a partial surprise to researchers and demonstrates the value of small, phase 1 clinical trials.

Fly Out 2010 Takes Off

“Sometimes change starts with the little things, like sharing your story with a member of Congress.”

These words, spoken by MDA Health Care Service Coordinator Carolyn McNutt after an August meeting with Arkansas Rep. Mike Ross, succinctly capture the purpose of the MDA Fly Out and make clear why the fledgling program is so successful.

Fly Out 2010 Slideshow

This year's Fly Out was a resounding success, with dozens of MDA families, sponsors and advocates meeting with members of Congress and their staffs in states and districts from coast to coast (and even in Hawaii).

In all of these meetings, which took place in the home districts of more than 30 U.S. senators and representatives throughout August and September, families shared their stories and asked for support for legislation important to the neuromuscular community.

The Future of Antisense: FDA, NIH Talk It Out

This story was updated Oct. 6, 2010.

Researchers, clinicians, pharmaceutical industry executives, and representatives from advocacy groups, including MDA, met in Washington Sept. 27-28, 2010, to discuss moving forward with antisense-based therapies for neuromuscular disease.

NIH Continues Funding for MD Research

The U.S. National Institutes of Health (NIH) announced Sept. 29, 2010, that it will allocate more than $4.5 million for the first year of a five-year commitment to explore new treatment strategies for various forms of muscular dystrophy.

Support will go to three U.S. institutions: Nationwide Children's Hospital in Columbus, Ohio; the University of Pennsylvania in Philadelphia; and the University of Iowa in Iowa City.

Overactive Protein Causes Motor Neuron Death in SBMA

Overactive function of normal androgen receptor protein and its interaction with disease-modifying "partner proteins" has been implicated as the specific underlying cause of motor neuron (nerve cell) degeneration and death in spinal-bulbar muscular atrophy (SBMA, or Kennedy disease).

Research Briefs: DMD, FA, DM, PM, IBM, MG, LEMS

Duchenne muscular dystrophy

Advocacy a Focus for New Ms. Wheelchair America with LGMD

McArthur was crowned Ms. Wheelchair American at the pageant in Grand Rapids, Mich., this August. (Glen Lipton Photography)

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