Recent therapeutic developments in spinal muscular atrophy

Creative Commons License

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 (SCI-Expanded) identifier identifier identifier

  • Publication Type: Article / Review
  • Volume: 48 Issue: 2
  • Publication Date: 2018
  • Doi Number: 10.3906/sag-1712-1
  • Journal Name: TURKISH JOURNAL OF MEDICAL SCIENCES
  • Journal Indexes: Science Citation Index Expanded (SCI-EXPANDED), Scopus, TR DİZİN (ULAKBİM)
  • Page Numbers: pp.203-211
  • Hacettepe University Affiliated: Yes

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.