Saturday 28 April 2012
Living with SMA
TUCSON, Ariz., April 25, 2012 /PRNewswire-USNewswire/ -- Repligen Corp., in Waltham, Mass., announced today that its experimental drug RG3039, designed to treat spinal muscular atrophy (SMA), was safe and well-tolerated in a phase 1 clinical trial.Avery was diagnosed with spinal muscular atrophy, a genetic disorder that attacks spinal neurons and progressively debilitates muscle function. Avery's was Type 1, the most severe kind, and doctors told them their little girl had only 18 months
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