Combination therapy with onasemnogene and risdiplam in spinal muscular atrophy type 1

Oechsel, KF; Cartwright, MS

Oechsel, KF (corresponding author), Wake Forest Sch Med, Dept Neurol, Winston Salem, NC 27157 USA.

MUSCLE & NERVE, 2021; 64 (4): 487

Abstract

Introduction/Aims There are currently three medications approved for spinal muscular atrophy (SMA), but the use of these medications in combination ha......

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