Quest Magazine

Intravenous AVI4658 Shows Safety, Benefit in DMD

The experimental drug AVI4658, in development by AVI BioPharma to treat Duchenne muscular dystrophy (DMD) caused by specific genetic mutations, was well tolerated and resulted in increased production of the needed dystrophin protein. Measured aspects of cardiac, pulmonary and skeletal muscle function remained stable.

Low-Dose Ataluren Shows Some Benefit in DMD/BMD

A low-dose regimen of ataluren (formerly called PTC124), an experimental drug developed by PTC Therapeutics to treat  Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD) caused by a certain type of genetic mutation, is superior to a high-dose regimen or a placebo.

ALS TDI Conference Summarizes ALS Research Progress

Themes discussed throughout the research symposium included ALS genetics, clinical trials, the role of the immune system in disease onset and progression, biomarkers, and the use of stem cells in ALS therapeutics.

Diaphragm Pacing System Awaiting FDA Approval for ALS

The Synapse Biomedical NeuRx Diaphragm Pacing System (DPS), a device that stimulates the respiratory diaphragm with electrical signals, has received Humanitarian Use Device designation from the U.S. Food and Drug Administration (FDA), allowing it to be used by people with amyotrophic lateral sclerosis (ALS) who have a “stimulatable” diaphragm and are experiencing chronic breathing problems.

Comic with BMD Builds Accessibility One Doorway at a Time

Jonah Bascle (rhymes with “rascal”) unleashes his zany brand of stand-up comedy on New Orleans nightclub and restaurant patrons four or five times a week. As an unusual consequence of these gigs, more and more of the historical city’s old buildings are becoming accessible to people in wheelchairs.

As a guy who uses a wheelchair himself due to Becker muscular dystrophy, Bascle, 24, knows first hand how difficult it is to navigate the entrances to many long-established buildings in the legendary home of Mardi Gras.

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.

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