Recent therapeutic developments in spinal muscular atrophy

Authors: GAMZE BORA, AYŞE YEŞBEK-KAYMAZ, CAN EBRU BEKİRCAN-KURT, VİLDAN GÖKNUR HALİLOĞLU, HALUK AYDIN TOPALOĞLU, HAYAT ERDEM-YURTER, SEVİM ERDEM-ÖZDAMAR

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.

Keywords: Spinal muscular atrophy, therapeutic approaches, clinical trials

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