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. , ...Daha Fazla

TURKISH JOURNAL OF MEDICAL SCIENCES, cilt.48, ss.203-211, 2018 (SCI İndekslerine Giren Dergi) identifier identifier identifier

  • Cilt numarası: 48 Konu: 2
  • Basım Tarihi: 2018
  • Doi Numarası: 10.3906/sag-1712-1
  • Dergi Adı: TURKISH JOURNAL OF MEDICAL SCIENCES
  • Sayfa Sayıları: ss.203-211

Özet

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.