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- W4239985280 abstract "In the phase 1 study (NCT02122952), AVXS-101, a potential SMN gene replacement therapy, improved survival and motor function in SMA1 patients. In contrast with natural history, increased motor function, measured by the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND), was detected as early as 1 month, and increased to levels that are not observed in the natural history of SMA1. Four patients experienced asymptomatic transient elevations in serum aminotransferase soon after dosing and were attenuated by prednisolone. We report study design and baseline demographics of STR1VE, a phase 3 study (NCT03306277) investigating efficacy and safety of AVXS-101. STR1VE is a multicenter (16 sites), open-label, single-arm, one-time-dose study in a minimum of 15 SMA1 patients <6 months of age (bi-allelic SMN1 mutations, 1-2xSMN2). For the first 3 patients, a 4-week dosing interval allowed review of safety and early signs of efficacy prior to dosing the next patient. Primary outcomes are independent sitting for 30 seconds at 18 months of age, and survival (avoidance of death/permanent ventilation) at 14 months. As of January 30, 2018, 3 SMA1 patients (2xSMN2) have been enrolled. Age at enrollment and symptom onset was 2.7 months (1.8-4.3 months) and 1.0 months (0.5-2.0 months). The mean baseline CHOP-INTEND score was 24.3 (18-34). No patients required ventilatory or nutritional support. The AVXS-101 phase 3 study investigates efficacy and safety of a one-time intravenous infusion of AVXS-101 in SMA1 patients. Updated patient data with follow-up will be presented." @default.
- W4239985280 created "2022-05-12" @default.
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- W4239985280 date "2018-10-01" @default.
- W4239985280 modified "2023-10-14" @default.
- W4239985280 title "SMA THERAPIES I" @default.
- W4239985280 doi "https://doi.org/10.1016/j.nmd.2018.06.209" @default.
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