Spinal Muscular Atrophy (SMA) is the leading genetic cause of death in infants and toddlers and is associated with a high rate of childhood mortality in its most severe forms. In a collaboration between Roche Pharma Research and Early Development (pRED), PTC Pharmaceuticals and the SMA Foundation, orally available molecules were discovered that may specifically correct a certain genetic defect in this devastating disorder. In mouse models of SMA, the compounds allow the animals to survive, grow and develop a normal neuromuscular system. We are working together to make a difference for SMA patients and their families.
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