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

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.

Keywords

Spinal muscular atrophy, therapeutic approaches, clinical trials

First Page

203

Last Page

211

Recommended Citation

BORA, GAMZE; YEŞBEK-KAYMAZ, AYŞE; BEKİRCAN-KURT, CAN EBRU; HALİLOĞLU, VİLDAN GÖKNUR; TOPALOĞLU, HALUK AYDIN; ERDEM-YURTER, HAYAT; and ERDEM-ÖZDAMAR, SEVİM (2018) "Recent therapeutic developments in spinal muscular atrophy," Turkish Journal of Medical Sciences: Vol. 48: No. 2, Article 1. https://doi.org/10.3906/sag-1712-1
Available at: https://journals.tubitak.gov.tr/medical/vol48/iss2/1