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Recent therapeutic developments in spinal muscular atrophy

Bora, Gamze; Ayse, Yesbek-Kaymaz; Can Ebru, Bekircan Kurt; Vildan Göknur, Haliloglu; Haluk Aydin, Topaloglu; Hayat, Erdem Yurter; Sevim, Erdem Özdamar.
Turk J Med Sci; 48(2): 203-211, 2018 Apr 30.
Artigo em Inglês | MEDLINE | ID: mdl-29714429
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.