MDA News and Updates

Moving Toward 'Next-Generation' Gene Therapy

May 22, 2013

Planning for the next generation of gene and stem cell therapies for muscular dystrophies — even as the first generation is still under development — was the theme of a joint symposium sponsored by MDA and the Association Française Contre les Myopathies (French Association Against Myopathies, or AFM) at the 16th annual me

More Data Reported from Neuralstem's Phase 1 Stem Cell Trial in ALS

May 21, 2013

Recently announced data from a phase 1 clinical trial of NSI-566 neural stem cells in people with amyotrophic lateral sclerosis (ALS) show that a subset of trial participants had better outcomes than the others.

New MDA/ALS Center Opens at LSU

May 20, 2013

The Muscular Dystrophy Association has designated the MDA clinic at Louisiana State University Health Sciences Center School of Medicine in New Orleans as an MDA/ALS center. This brings the total number of these specialized clinics to 44.

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.

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

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.

26th Annual MDA Ride for Life Breaks $1 Million Mark to Help Fight Muscle Disease

May 10, 2013

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.

DMD UPDATE: Drisapersen Outperforms Placebo in Walking Test

May 08, 2013

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.

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.

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.

Lowe's Raises $8.8 Million for MDA's Shamrocks Campaign

May 02, 2013

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.

MDA Scientific Conference Blog: Playing in the Same Sandbox

April 25, 2013

MDA Scientific Conference Blog: We Need Each Other

April 25, 2013

‘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.