Impact of age and motor function in a phase 1/2A study of infants with SMA Type 1 receiving single-dose gene replacement therapy

Linda P. Lowes,Lindsay N. Alfano,W.David Arnold,Richard Shell,Thomas W. Prior,Markus McColly,Kelly J. Lehman,Kathleen Church,Douglas M. Sproule,Sukumar Nagendran,Melissa Menier,Douglas E. Feltner,Courtney Wells,John T. Kissel,Samiah Al-Zaidy,Jerry Mendell

Pediatric Neurology（2019）

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摘要

The rapid, significant motor improvements among infants with severe SMA1 treated with AVXS-101 at an early age highlight the importance of newborn screening and early treatment and demonstrate the therapeutic potential of AVXS-101 regardless of baseline motor function.

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关键词

AVXS-101,Gene replacement therapy,Spinal muscular atrophy,CHOP-INTEND,Onasemnogene abeparvovec

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