MDA News and Updates

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How to Obtain Durable Medical Equipment Via Medicare
January 23, 2013
Durable medical equipment (DME), such as wheelchairs, walkers, portable oxygen devices and hospital beds, can be very expensive — and the process for obtaining them from Medicare can be very confusing.  Learn about Medicare coverage of DME and get the latest information on obtaining such equipment in a webinar Tuesday, Jan. 29, from 2 to 3 p.m. Eastern time.  
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MDA Establishes Myotonic Dystrophy Clinical Research Network
January 23, 2013
MDA has launched a new, five-center clinical research network focused on type 1 and type 2myotonic muscular dystrophy (MMD1 and MMD2, also known as DM1 and DM2), with the principal goal of preparing for testing of new MMD treatments as they become available.
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Spice for CMT1B? Curcumin Shows Benefit in Research Mice
January 20, 2013
Mice with a disorder resembling the type 1B form of Charcot-Marie-Tooth disease (CMT) benefited from treatment with either of two forms of oral curcumin, a component of the spice turmeric, according to researchers supported in part by MDA.
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Friedreich’s Ataxia: Drug Enters Phase 2b Clinical Trial
January 15, 2013
Edison Pharmaceuticals has launched a phase 2b clinical trial of its experimental drug EPI-743 in adults with Friedreich's ataxia (FA) to assess whether the drug has positive effects on visual function, neurological and neuromuscula
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DMD: Dystrophin-Deficient Dogs Benefit from Gene Therapy
January 15, 2013
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.
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Podcast: What Happened with Dexpramipexole for ALS?
January 15, 2013
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MTM Mice Respond to Modified Myotubularin Protein Injections
January 10, 2013
Update (Jan. 10, 2013):This story has been updated with additional information about MDA funding of this and related research projects.
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ALS Stem Cell Trial in Israel Is ‘Accelerated’
January 09, 2013
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Pfizer and Repligen to Collaborate on SMA Therapy Development
January 04, 2013
The biotechnology firm Repligen today announced it has entered into an agreement with the global pharmaceutical company Pfizer to advance Repligen’s spinal muscular atrophy (SMA)
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Phase 3 Trial of Dexpramipexole in ALS Fails to Show Benefit
January 02, 2013
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).
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DMD, BMD: Nationwide Children's Podcast Explores Dystrophin's Interactions
December 30, 2012
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
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MD Briefs: Gene Therapy, Exon Skipping, Stem Cells
December 26, 2012
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.
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Federal SMA Project Issues Progress Report
December 23, 2012
A small-molecule drug candidate for spinal muscular atrophy (SMA) and an effective strategy for advancing therapy development are the primary outcomes being reported by the Spinal Muscular Atrophy (SMA) Project, a program established in 2003 by the
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ALS Briefs from the 2012 ALS/MND Symposium
December 18, 2012
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.
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Second Mouse Model of Periodic Paralysis Developed
December 16, 2012
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 blo
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ALS Briefs: Clinical Trial Updates at the 2012 ALS/MND Symposium
December 13, 2012
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.
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Tirasemtiv Shows Promise in MG
December 12, 2012
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.
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DMD: Eteplirsen Results Still Strong at 62 Weeks
December 07, 2012
Eteplirsen, an exon-skipping drug in development by Sarepta Therapeutics to treat Duchenne muscular dystrophy (DMD) caused by specific mutations in the dystrophin gene, continues to show benefit at 62 w
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What’s Next for Eteplirsen? Sarepta Discusses FDA Approval and Future Trials
December 07, 2012
In both October and December 2012, Sarepta Therapeutics announced very encouraging results from a 12-person phase 2b trial of eteplirsen, an exon-skipping compound that is a potential treatment for Duchenne muscular dystroph
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MDA Building ‘Transitional Freedom’ Partnerships
December 06, 2012
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