NINDS

Gene therapy delivered to muscles reaches nerve cells; collaboration aims to advance gene therapy development program; a mouse model represents milder forms of the disease

posted on March 14, 2013 - 4:51pm
Spinal muscular atrophy (SMA) is a disease in which nerve cells that control muscles (motor neurons) in the spinal cord die, causing progressive weakness in the voluntary muscles. Recent research news includes advances in delivery methods for gene therapy treatment of SMA, and creation of a new mouse model that could help scientists better understand and develop treatments for the disease....

The National Institutes of Health has launched a biomarkers and natural history study in infants with type 1 spinal muscular atrophy

posted on September 18, 2012 - 10:25am
Update (March 28, 2013): This story was updated with a link to the ClinicalTrials.gov site for this study.

New imaging techniques illuminate muscle and nerve, speeding research and aiding clinical trials

posted on July 1, 2012 - 3:09pm
QUEST Vol. 19, No. 3
Researchers are very good at understanding what is going on with things they can’t see directly, whether it be the membranes of muscle cells or the motor neurons of the spinal cord. But what is true for the rest of us is equally true for scientists: Seeing something is often the quickest and most direct way to understand it.

Participants will be randomly assigned to a strength or stretching exercise program, and undergo multiple evaluations

posted on August 9, 2011 - 12:00am
Update June 19, 2013: This study is nearing completion. However, enrollment is still open and additional participants are needed.