- Turkish Journal of Medical Sciences
- Vol: 48 Issue: 2
- Recent therapeutic developments in spinal muscular atrophy
Recent therapeutic developments in spinal muscular atrophy
Authors : Gamze Bora, Ayşe Yeşbek-kaymaz, Can Ebru Bekircan-kurt, Vildan Göknur Haliloğlu, Haluk Aydin Topaloğlu, Hayat Erdem-yurter, Sevim Erdem-özdamar
Pages : 203-211
View : 26 | Download : 9
Publication Date : 9999-12-31
Article Type : Makaleler
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