Mengnan Lu

Apr 14, 2024 | onlinelibrary.wiley.com | Mengnan Lu |Xueying Wang |Na Sun |Shaoping Huang

1 INTRODUCTION Spinal muscle atrophy (SMA) is a neurogenic muscle disease characterized by motor neuron degeneration and severe muscular atrophy.1 SMA is an autosomal recessive disease caused by a mutation in the surviving motor neuron gene (SMN1) on the long arm of chromosome 5 (5q13.1).