MDA News and Updates

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FA: RG2833 is Well-Tolerated and Increases Frataxin Gene Activity
May 17, 2013
Interim results from a phase 1 clinical trial of RG2833 in people with Friedreich’s ataxia (FA) show that the experimental drug is well-tolerated, and that it appears to increase the activity of the gene for the frataxin protein.
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Newborn Screening Recommended for Pompe Disease
May 17, 2013
The Discretionary Advisory Committee on Heritable Disorders in Newborns and Children (DACHDNC) today voted to add Pompe disease (acid maltase deficiency) to a
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Trial Tests 'Master Regulator' in ALS
May 16, 2013
A phase 2 clinical trial to test the experimental drug GM604 in people with amyotrophic lateral sclerosis (ALS) has opened at two trial sites in the United States. Enrollment is expected to begin soon.
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26th Annual MDA Ride for Life Breaks $1 Million Mark to Help Fight Muscle Disease
May 10, 2013
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Melatonin is Neuroprotective in ALS Mice
May 10, 2013
Melatonin, a naturally occurring hormone produced by the pineal gland, is best known for its role in regulating sleep.
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DMD UPDATE: Drisapersen Outperforms Placebo in Walking Test
May 08, 2013
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DMD, BMD: Phase 3 Trial Opens for Stop Codon Read-Through Drug
May 08, 2013
A large-scale, multinational phase 3 trial of the experimental drug ataluren has opened its first trial site, in Cincinnati, Ohio.
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SMA: Trial Tests Antisense Therapy in Infants
May 07, 2013
A phase 2 clinical trial to test multiple doses of the experimental drug ISIS-SMNRx in infants with spinal muscular atrophy (SMA) has opened at four trial sites in the United States and Canada. Enrollment is expected to begin soon.
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MTM Phone-Based 'Event' Study Now Open
May 03, 2013
The Myotubular Myopathy Event Study, a telephone-based survey, will gather information about MTM-associated events, such as emergency room visits, hospitalizations, medication reactions, and complications from medical procedures, as well as improved or declined motor and respiratory function.
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Lowe's Raises $8.8 Million for MDA's Shamrocks Campaign
May 02, 2013
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UPDATE: ACE-031 Clinical Trials in Duchenne MD
May 02, 2013
Biopharmaceutical companies Acceleron Pharma and Shire announced April 21, 2011, that MDA-supported clinical trials of ACE-031 for Duchenne muscular dystrophy (DMD) have been halted.
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MDA Scientific Conference Blog: We Need Each Other
April 25, 2013
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MDA Scientific Conference Blog: Playing in the Same Sandbox
April 25, 2013
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‘Focused, Intense’ MDA Conference Advances Neuromuscular Disease Research
April 25, 2013
Turning neuromuscular disease research into treatments as quickly and effectively as possible was the overarching theme of dozens of formal presentations, nearly 200 scientific posters, and countless informal conversations at the MDA Scientific Conference, April 21-24.
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MDA Scientific Conference Blog: Gracious Professionalism and ‘Coopertition’
April 24, 2013
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MDA Scientific Conference Blog: First Things First: The Necessity and Challenges of Mouse Models
April 24, 2013
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MDA Scientific Conference Blog: Something To Talk About
April 23, 2013
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MDA Scientific Conference Blog: Seeds Into Gardens — An Old Hand’s Perspective
April 23, 2013
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Harley-Davidson And MDA Celebrate 20 Years Of Chrome And Leather At Annual Black-N-Blue Ball
April 23, 2013
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Modifying Cellular Stress Response as a Way To Treat ALS
April 23, 2013
An MDA-supported research team has identified a series of compounds that appear to work both alone and in combination to protect the muscle-controlling nerve cells (motor neurons) that are lost in amyotrophic lateral sclerosis (ALS).
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