Nusinersen for the treatment of spinal muscular atrophy

Chiriboga, CA

Chiriboga, CA (reprint author), Columbia Univ, SMA Clin Res Ctr, Div Pediat Neurol, Dept Neurol,Med Ctr, 180 Ft Washington Ave, New York, NY 10027 USA.

EXPERT REVIEW OF NEUROTHERAPEUTICS, 2017; 17 (10): 955

Abstract

Introduction: Spinal muscular atrophy (SMA) is an autosomal recessive degenerative neuromuscular disorder characterized by loss of spinal motor neuron......

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