A clinical trial to test a common antibiotic as a treatment for another form of muscular dystrophy is slated to start by year-end. As a result of these and other promising research initiatives, the 1999 Jerry Lewis MDA Telethon for the Muscular Dystrophy Association will celebrate rapid scientific progress. We're awaiting final National Institutes of Health review of the protocol and expect, in September, to begin the gene therapy trial already authorized by the Food and Drug Administration. This gene therapy safety trial for limb-girdle muscular dystrophy is the first for a neuromuscular disease.
Gene therapy involves replacement of disease-causing genes with correctly functioning ones.
The approach may eventually lead to cures for many genetic diseases. In July, MDA-funded investigators found that gentamicin, an antibiotic commonly used for bacterial infections, improved muscle function and reversed signs of muscular dystrophy in mice. This year's Telethon, to be broadcast Sept. 5-6, will support MDA programs to benefit those affected by 40 neuromuscular diseases in the upcoming year. I'm deeply moved by the caring response millions give the Telethon each year. Consistently ranked among leading national voluntary health agencies for the level of income expended on program services, almost all of MDA's income is derived from private voluntary contributions.