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Turkish Journal of Medical Sciences

DOI

10.3906/sag-1712-1

Abstract

Proximal spinal muscular atrophy (SMA) is an inherited neurodegenerative disease with a heterogeneous clinical phenotype. Although there is no cure for SMA, several strategies are currently being developed. In this review, we summarize the ongoing clinical trials and molecular mechanisms of successful approaches to SMA treatment.

First Page

203

Last Page

211

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