Five-year disease-modifying therapeutic experience of 102 Chinese pediatric 5q-spinal muscular atrophy: a retrospective analysis
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Overview
Retrospective analysis reports a 97.08% survival rate in pediatric patients with 5q-SMA receiving nusinersen treatment, highlighting complications like scoliosis and respiratory infections.
Key Points
Motor-function gains were statistically significant, but complications remained common, including malnutrition and fractures.
Survival rate reached 97.08% over five years in 102 pediatric patients treated with nusinersen and other DMTs.
Increased IGF-1 levels were observed in SMA types 1 and 2, but lacked statistical significance during treatment.
Longer diagnostic to treatment intervals correlated with poorer motor function outcomes and higher complication rates.