As the scientific community has developed a better understanding of the biological processes leading toward neuromuscular disease, MDA has also broadened its funding strategy into “translational” research. Translational research covers the work necessary to develop a potential therapeutic from the point when a potential drug has been identified to the stage in which the candidate therapy must be tested in humans (clinical trials). This includes improving the compound, testing to see if it is safe and effective in animal disease models, determining appropriate doses, and other tests required by the Food and Drug Administration (FDA) before a drug can be tested in humans. Once the best, or “lead” compound is identified, this work is also called “preclinical research”. In 2003, MDA launched its Translational Research Program specifically aimed at accelerating this area of research.
MDA Venture Philanthropy (MVP) is MDA’s flagship program for drug discovery. Bridge to Industry is a new pilot program aimed at supporting the training of translational research scientists.
Muscular Dystrophy Association
Heidi Pottinger, M.A., M.P.H.
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