Spinal Muscular Atrophy (SMA)

SMA: Full Speed Ahead

A Closer Look: SMA Slideshow

MDA Awards $8.5 Million to 31 Neuromuscular Disease Research Projects

In its summer 2013 round of research grant awards, the Muscular Dystrophy Association aims to catalyze research progress in a dozen neuromuscular diseases, with an eye toward applying that knowledge to related muscle diseases, as well.

“A large number of our grants are investigating new therapeutic technologies,” notes Jane Larkindale, MDA's vice president of research. “These are 'platform' technologies, where successes can be transferred well beyond the specific disease in which they are developed and tested.”

SMA — Bennett Novitch, Ph.D.

Bennett Novitch, assistant professor of neurobiology at the University of California, Los Angeles, was awarded an MDA research grant totaling $300,000 over a period of three years to study the development of motor neurons that control respiration and their significance for spinal muscular atrophy (SMA).

SMA — Umrao Monani, Ph.D.

Umrao Monani, associate professor at Columbia University Medical Center in New York City, was awarded an MDA research grant totaling $300,000 over a period of three years to study how junctions between neurons and muscle are affected in spinal muscular atrophy (SMA).

SMA Briefs: Treatment Window, Celecoxib

Recent spinal muscular atrophy (SMA) research includes findings that shed additional light on the optimal "window of opportunity" for treatment and point toward a potential candidate for therapeutic development.

SMA: Trial Tests Antisense Therapy in Infants

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.

‘Focused, Intense’ MDA Conference Advances Neuromuscular Disease Research

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.

A palpable sense of excitement pervaded the sold-out event thanks to the unprecedented number of experimental treatments in clinical trials for neuromuscular diseases, and the unique opportunity the conference provided for information-sharing and collaboration among scientific professionals from many disciplines.

MDA Scientific Conference To Emphasize Therapy Development

The Muscular Dystrophy Association’s annual conference being held in Washington, D.C., on April 21-24, 2013, is centered on the theme Therapy Development for Neuromuscular Diseases: Translating Hope into Promise.

SMA Research Briefs: Testing Drugs from Other Diseases

In the search for therapies for spinal muscular atrophy (SMA), researchers are testing two drugs with connections to other neuromuscular disorders: riluzole, which is approved for use in amyotrophic lateral sclerosis (ALS); and tirasemtiv, which is already in testing for ALS and

SMA Antisense Drug Shows Safety, Tolerability in Phase 1 Trial

An experimental drug designed to treat the underlying molecular defect in spinal muscular atrophy (SMA) has shown encouraging results in a phase 1 trial.