Treatment of SMA type 1 infants using a single-dose AAV9-mediated gene therapy via intrathecal injection of GC101: An open-label, single-arm study

The patients were recruited in the study and received GC101 treatment between January 25, 2022 and April 12, 2022 at the Seventh Medical Center of PLA General Hospital (Beijing, China) [Figure 1A]. [4,5] Atorvastatin was used in P2 (5th–6th weeks) because of the consistently high level of LDL cholesterol and it was considered to be related to the elevated levels of AST (6th week) and ALT (6th week) [Supplementary Figure 3, http://links.lww.com/CM9/C76]. [...]our single-dose AAV9-mediated gene therapy of GC101 by intrathecal delivery for SMA type 1 patients proved to be safe and could extend survival time and improve motor function than in natural history of SMA patients. A proposal with detailed description of study objectives and statistical analysis plan will be needed for the evaluation of the reasonability of requests.