Effectiveness of gene therapy for motor function in children with spinal muscular atrophy: a systematic review and meta-analysis of randomized controlled trials

Abstract

Review question / Objective: To identify the efficacy of gene therapy for motor function in children with spinal muscular atrophy (SMA); to compare effects and safety of potential different treatments for SMA. Condition being studied: Spinal muscular atrophy (SMA) is a neurodegenerative disorder caused by mutations in SMN1 (encoding survival motor neuron protein (SMN)). Reduced expression of SMN leads to loss of α-motor neurons, severe muscle weakness and often early death. Estimated incidence is 1 in 6,000 to 1 in 10,000 live births and carrier frequency of 1/40-1/60. INPLASY 1 International Platform of Registered Systematic Review and Meta-analysis Protocols INPLASY PROTOCOL Effectiveness of gene therapy for motor function in children with spinal muscular atrophy: a systematic review and metaanalysis of randomized controlled trials Chen, B 1 ; Zhou, TT 2 ; Gong, YL 3 . To cite: Chen et al. Effectiveness of gene therapy for motor function in children with spinal muscular atrophy: a systematic review and metaanalysis of randomized controlled trials. Inplasy protocol 202350072.