A phase I, single-site, dose escalation study to evaluate the safety and efficacy of gene transfer therapy in infants with for Spinal Muscular Atrophy Type 1 (SMA1) who are 9 months old or younger has opened at Nationwide Children’s Hospital in Columbus, Ohio. A total of nine patients will be randomly assigned to receive either a low dose or a high dose of gene transfer infusion, as a one-time intravenous injection. Patients will continue to be monitored at Nationwide Children’s Hospital including physical exams and blood tests for two years after gene transfer.
