Quest Magazine

DMD: Dystrophin-Deficient Dogs Benefit from Gene Therapy

For the first time, gene therapy using a highly miniaturized dystrophin gene resulted in significant improvement in muscle structure and function in dogs with a disorder mimicking human Duchenne muscular dystrophy.

Podcast: What Happened with Dexpramipexole for ALS?

MTM Mice Respond to Modified Myotubularin Protein Injections

Update (Jan. 10, 2013):This story has been updated with additional information about MDA funding of this and related research projects.

Phase 3 Trial of Dexpramipexole in ALS Fails to Show Benefit

Biotechnology company Biogen Idec today announced disappointing results from its phase 3 trial of dexpramipexole, an experimental drug the company has been developing to treat amyotrophic lateral sclerosis (ALS).

DMD, BMD: Nationwide Children's Podcast Explores Dystrophin's Interactions

In a December 2012 podcast from Nationwide Children's Hospital, cell biologist Federica Montanaro discusses her team's recent progress in understanding how various proteins interact with dystrophin and how these interactions differ in the heart versus the skeletal muscles.

MD Briefs: Gene Therapy, Exon Skipping, Stem Cells

Update (Jan. 23, 2013):The "Building better utrophin" section was updated to reflect the availability of a Jan. 22, 2013, press release from the University of Missouri.

Below is a wrap-up of recent research news about the development of therapies for Duchenne, Becker and limb-girdle muscular dystrophies.

ALS Briefs from the 2012 ALS/MND Symposium

The 23rd International Symposium on ALS/MND (motor neuron disease), held in Chicago Dec. 5-7, 2012, brought together more than 900 researchers, clinicians and other health care professionals from 30 countries to hear presentations on the latest in ALS care and research.

Second Mouse Model of Periodic Paralysis Developed

Scientists supported in part by MDA have developed a second type of research mouse with a disorder mimicking hypokalemic periodic paralysis, a genetic disorder in which recurrent attacks of weakness or paralysis occur in association with low potassium levels in the bloodstream. Like the majority of humans with this disorder, the mice have a mutation in the gene for a muscle-fiber calcium channel, a key player for the initiation of muscle contraction.

ALS Briefs: Clinical Trial Updates at the 2012 ALS/MND Symposium

The 23rd International Symposium on ALS/MND (motor neuron disease), held in Chicago Dec. 5-7, 2012, brought together more than 900 researchers, clinicians and other health care professionals from 30 countries to hear presentations on the latest in ALS care and research.

Tirasemtiv Shows Promise in MG

The experimental drug tirasemtiv has shown promise in a phase 2a clinical trial in 32 people with myasthenia gravis (MG). Improvements in general and respiratory muscle function occurred in response to the medication.

Six hours after receiving a single, oral dose of tirasemtiv, study participants showed improvements on a standardized scale of MG severity (the Quantitative MG score) and in forced vital capacity, a measurement of respiratory function.

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