Tuesday, December 20, 2011
Isis Pharmaceuticals Inc., of Carlsbad, Calif., initiated a Phase I study of ISIS-SMNRx in patients with spinal muscular atrophy (SMA). The single-dose, dose-escalation study is designed to assess the safety, tolerability and pharmacokinetic profile of the drug in children with SMA between the ages of 2 and 14 who are medically stable. ISIS-SMNRx will be administered intrathecally as a single injection directly into the spinal fluid.
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