Spinal Muscular Atrophy (SMA)

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.

Research Briefs: Spinal Muscular Atrophy

Skeletal muscle damage reversed in SMA mice

In addition to muscle damage caused by the loss of motor neurons in the spinal cord, skeletal muscle degeneration inspinal muscular atrophy (SMA) also stems from low levels of the protein SMN in muscle fibers. Now a team of scientists from Germany and the United Kingdom reports that muscle damage caused by low SMN can be reversed with drugs.

Induced Stem Cells Require Cautious Approach

Stem cells have been much in the news lately, including for neuromuscular diseases.

Tabi Estrellado: Writing Code, Writing Songs, Winning Awards

Tabitha Estrellado is living her dream. She’s a 27-year-old career woman with a nice apartment in Manhattan. She loves her job, has a sweet commute, and writes music and performs locally in her spare time.

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