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Dergiler / Turkish Journal of Medical Sciences / 2018 / Cilt: 48 - Sayı: 2

Recent therapeutic developments in spinal muscular atrophy

Gamze BORA, Haluk Aydın TOPALOĞLU, Can Ebru BEKİRCAN KURT, Ayşe YEŞBEK KAYMAZ, Vildan Göknur HALİLOĞLU, Hayat ERDEM YURTER, Sevim ERDEM ÖZDAMAR

Sayfa
203–211
DOI
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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 clinicaltrials and molecular mechanisms of successful approaches to SMA treatment.