Abstract
To assess the value of onasemnogene abeparvovec with respect to outcomes in patients with spinal muscular atrophy type 1 (SMA1). SMA1 is characterized by motor neuron degeneration and progressive muscle weakness. Untreated SMA is the leading genetic cause of infant mortality. Outcomes of the 22 patients with SMA1 (2XSMN2) treated with onasemnogene abeparvovec in the phase 3 STR1VE study (NCT03306277) were compared with those of untreated patients in 2 natural history studies (PNCR: ≤36 months, n=23; NN101: ≤24 months, n=16) and the ENDEAR (NCT02193074) control group (n=27).
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