DMD: 'Permanent' Gene Repair Strategy Looks Good in Lab

DMD: 'Permanent' Gene Repair Strategy Looks Good in Lab

A strategy to repair DNA in cells taken from boys with Duchenne muscular dystrophy has resulted in production of dystrophin protein molecules

Genzyme and MDA Provide Pompe Families with Free Testing

Genzyme and MDA Provide Pompe Families with Free Testing

The biopharmaceutical company will provide first-step Pompe testing (measuring GAA levels in blood) if an MDA clinic physician suspects the disorder

MD Briefs: Registries Are Still Open

MD Briefs: Registries Are Still Open

Disease registries are seeking participants with CMD, FSHD, LGMD, MMD and Pompe disease

Study Suggests Sleep-Disordered Breathing Common in FA

Study Suggests Sleep-Disordered Breathing Common in FA

Nighttime respiratory problems may be more common in people with Friedreich's ataxia than in the general population, necessitating the need for regular screening

MTM: MDA Funds Development of Myotubularin-Based Treatment

MTM: MDA Funds Development of Myotubularin-Based Treatment

A $1.2 million MDA grant will help move an experimental cell-penetrating treatment for myotubular myopathy toward human testing

SBMA: Mixed Results in Study of Clenbuterol

SBMA: Mixed Results in Study of Clenbuterol

Results from a 20-person pilot trial suggest that clenbuterol may improve motor and respiratory function in people with spinal-bulbar muscular atrophy, but safety concerns were raised

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How the FDA tries to balance urgent needs against real risks when evaluating new drugs

posted on April 1, 2013 - 9:25am
The approval of new drugs in the United States is a complex process that seeks to balance the need to protect the health and safety of the population with the need to provide treatment as soon as possible for serious or life-threatening disorders.

MDA advocacy in Washington

posted on April 1, 2013 - 9:23am
I have had the great privilege of witnessing some of the most transformative legislative moments in our MDA community’s history. While we have many heroes and legends among us, there are also many heroes whose names most will never know — people who, through their outreach to their elected officials, brought great champions to the cause of neuromuscular disease research and services.

What’s ready now and what’s in your future

posted on April 1, 2013 - 9:21am
Entering into the world of disability should come with a giant neon sign that reads “Warning: Technology Ahead.” It’s inescapable. Not only is it all around us but for many of us, technology keeps us alive well beyond what the naysayers predict. Different neuromuscular diseases progress at different rates, but eventually we all start losing mobility, strength and/or dexterity. Some of us will...

On being treated as a person first during hospitalizations

posted on April 1, 2013 - 9:19am
MJ Purk Individuals with neuromuscular disorders oftentimes spend a great deal of time in the hospital and even more time interacting with those who work in the medical profession. In their interactions with medical personnel, some individuals are lucky enough to experience more than the expected amount of caring.

The right cushion makes all the difference in wheelchair comfort

posted on April 1, 2013 - 9:17am
Anyone using a wheelchair, even for a few hours a day, needs to sit on a cushion. The most obvious reason for using a wheelchair cushion is simply to offer general comfort by reducing friction and vibration. A good cushion also can wick away moisture and keep you cool.
posted on April 1, 2013 - 9:15am
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