This study will be recruiting shortly, and is open to qualifying Type 1 Infants. Canadian location is Toronto Hospital for Sick Children.
From FSMA-US: ISIS Pharmaceuticals Initiates Clinical Study in Infants with Spinal Muscular Atrophy. (April 23, 2013) Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced the initiation of a Phase 2 study of ISIS-SMNRx in infants with spinal muscular atrophy (SMA). The study, which will begin enrolling patients soon, is a Phase 2 study in eight patients with infantile-onset SMA. The study, which initiates the Phase 2/3 program for ISIS-SMNRx in infants, is designed to provide data to allow Isis to define the optimal dose for the larger planned Phase 2/3 study in infants and to provide safety and tolerability data. See more information...
Location and Recruiting Contact Information: A Study to Assess the Safety and Pharmacokinetics of ISIS SMNRx in Infants With Spinal Muscular Atrophy. This study will test the safety, tolerability, and pharmacokinetics of multiple doses of ISIS-SMNRx administered into the spinal fluid three times over the duration of the trial, in patients with Infantile-Onset Spinal Muscular Atrophy. See more information...
ISIS Pharmaceuticals Press Release
Posted April 26, 2013