A96 BEST OF PEDIATRICS: Avxs-101 Phase 1 Gene Therapy Clinical Trial In Sma Type 1: Interim Data Demonstrates Improvements In Survival, Motor Function, And Decreased Dependence On Ventilator Support

Here we report interim data on the survival, motor function, and supportive care use from an ongoing clinical trial of SMA1 infants treated with AVXS-101, a gene therapy. CONCLUSIONS In contrast with the published natural history, a single intravenous administration of AVXS-101 appears to demonstrat...

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Bibliographic Details
Published in:American journal of respiratory and critical care medicine 2017-01, Vol.195
Main Authors: Shell, R, Al-Zaidy, S, Arnold, W D, Rodino-Klapac, L, Prior, T W, Kotha, K, Lowes, L, Alfano, L, Berry, K, Church, K, Kissel, J, Nagendran, S, L'Italien, J, Sproule, D, Du, M, Cardenas, J, Burghes, A, Foust, K D, Meyer, K, Likhite, S, Kaspar, B K, Mendell, J R
Format: Article
Language:English
Subjects:
Clinical trials
Gene therapy
Pediatrics
Respiratory therapy
Ventilation
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Summary:Here we report interim data on the survival, motor function, and supportive care use from an ongoing clinical trial of SMA1 infants treated with AVXS-101, a gene therapy. CONCLUSIONS In contrast with the published natural history, a single intravenous administration of AVXS-101 appears to demonstrate a positive impact on the survival of both dosing cohorts and a dramatic, sustained impact on motor function in Cohort 2.
ISSN:1073-449X
1535-4970