Recent therapeutic developments in spinal muscular atrophy


BORA G. , YEŞBEK KAYMAZ A. , Bekircan Kurt C. E. , HALİLOĞLU V. G. , TOPALOĞLU H. A. , Erdem Yurter H. , ...More

TURKISH JOURNAL OF MEDICAL SCIENCES, vol.48, no.2, pp.203-211, 2018 (Journal Indexed in SCI) identifier identifier identifier

  • Publication Type: Article / Review
  • Volume: 48 Issue: 2
  • Publication Date: 2018
  • Doi Number: 10.3906/sag-1712-1
  • Title of Journal : TURKISH JOURNAL OF MEDICAL SCIENCES
  • Page Numbers: pp.203-211

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.