Spinal Muscular Atrophy (SMA)

Entrepreneur with SMA Starts Accessible Car Company

Stacy Zoern has entered yet a new phase in her already eventful life — this time at 25 miles per hour.

SMA Gene Associated with Sporadic ALS

Duplications (extra copies) of the SMN1 gene are a "major" risk factor for developing sporadic (noninherited) ALS (amyotrophic lateral sclerosis), a team of scientists based in the Netherlands and United Kingdom has reported.

MDA Commits $12 Million to Neuromuscular Disease Research

The Muscular Dystrophy Association has awarded 38 new grants totaling more than $12 million to fund research projects focused on its continuing mission to uncover the causes of, and develop therapies for, the more than 40 neuromuscular diseases in its program.

MDA's Board of Directors reviewed and approved the new grants based on recommendations from the Association's Scientific and Medical Advisory Committees, and the grants took effect Feb. 1.

SMA - Christian Lorson, Ph.D.

MDA awarded a research grant totaling $381,582 over a period of three years to Christian Lorson, a professor in the departments of veterinary pathobiology, and molecular microbiology & immunology, at the University of Missouri in Columbia. The funds will help support Lorson’s research into targeting skeletal muscle as a therapeutic strategy in spinal muscular atrophy (SMA).

SMA - Kentaro Sahashi, M.D., Ph.D.

Kentaro Sahashi, a postdoctoral research scientist at the Cold Spring Harbor Laboratory in Cold Spring Harbor, N.Y., was awarded an MDA development grant (DG) totaling $180,000 over a period of three years to study the roles of the SMN protein in spinal muscular atrophy (SMA).

SMA - Tomoyuki Awano, Ph.D.

Tomoyuki Awano, a postdoctoral research scientist in the department of pathology and cell biology at Columbia University Medical Center in New York, was awarded an MDA development grant (DG) totaling $180,000 over a period of three years to search for genes that modify the onset and disease course of spinal muscular atrophy (SMA).

MDA Funds Development of Utrophin 'Magnet' for DMD/BMD


Phase 1 Trial Opens of ISIS-SMNRx in Children with SMA

A 24-person, phase 1 trial to test the safety and tolerability of the experimental drug ISIS-SMNRx in children with spinal muscular atrophy (SMA) is now open at Columbia University Medical Center in New York, with additional sites expected to open in Boston, Philadelphia, Dallas and Salt Lake City.

PTC, Roche Team Up Against SMA

Swiss biotechnology company Roche, and PTC Therapeutics in South Plainfield, N.J., on Nov. 29, 2011, announced a collaboration through which the two companies will work together to advance drug development for spinal muscular atrophy(SMA).

Systemic Antisense Injections Rescue Severe SMA Mice

Treatment of a mouse model of severe spinal muscular atrophy (SMA) with an antisense oligonucleotide results in greater and longer-lasting benefit when given systemically than when given only to the central nervous system, new research shows.