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Safety and efficacy findings in the first-in-human trial (FIH) of the oral splice modulator branaplam in type 1 spinal muscular atrophy (SMA): interim results

Read time: 1 mins
Published:1st Oct 2017
Author: Charnas L, Voltz E, Pfister C, Peters T, Hartmann A, Berghs-Clairmont C et al.
Availability: Pay for access, or by subscription
Ref.:Neuromuscul Disord. 2017;27(S2):S207�S208
DOI:10.1016/j.nmd.2017.06.411
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