Spinal Muscular Atrophy (SMA) in the Therapeutic Era

Volume: 7, Issue: 3, Pages: 162 - 167
Published: Sep 1, 2019
Abstract
Spinal muscular atrophy (SMA) is an autosomal recessive disorder caused by a mutation in the SMN1 gene. It is relatively common worldwide, affecting approximately 1 in 11,000 live births, and about 1 in every 54 individuals is a carrier. The FDA-approved nusinersen (Spinraza) in December 2016 and onasmenogene abeparvovec (Zolgensma) in May 2019 for treatment of SMA after clinical trials showed slowed progression, improved motor function, and...
Paper Details
Title
Spinal Muscular Atrophy (SMA) in the Therapeutic Era
Published Date
Sep 1, 2019
Volume
7
Issue
3
Pages
162 - 167
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