Lab-made enzyme approved by FDA
Myozyme, a laboratory-engineered enzyme patented by Genzyme and developed in part with basic research funded by MDA, is approved for use in children and adults with acid maltase deficiency (Pompe disease). It replaces the missing enzyme in this metabolic muscle disease.
Gene therapy trial for Duchenne dystrophy begins
Scientists and physicians launch the first U.S. human gene therapy trial directed at Duchenne muscular dystrophy, with the support of a $1.6 million grant from MDA. The first of six boys with DMD receives an injection of genes for dystrophin, the missing protein in DMD, in one arm and a placebo in the other. The scientists will later measure dystrophin production and monitor the effects of the gene transfer on the children.
Variants in ‘detox’ genes found to raise ALS risk
MDA-supported investigators identify variations in and around genes known as PONs, whose normal role is to detoxify poisons such as pesticides and nerve gas, as risk factors for developing amyotrophic lateral sclerosis. The finding may help explain why Gulf War veterans have a higher than normal rate of ALS development and why occupational clusters of ALS (Lou Gehrig's disease) occasionally have been identified.