Recent therapeutic developments in spinal muscular atrophy

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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, sa.2, ss.203-211, 2018 (SCI-Expanded) identifier identifier identifier

  • Yayın Türü: Makale / Derleme
  • Cilt numarası: 48 Sayı: 2
  • Basım Tarihi: 2018
  • Doi Numarası: 10.3906/sag-1712-1
  • Dergi Adı: TURKISH JOURNAL OF MEDICAL SCIENCES
  • Derginin Tarandığı İndeksler: Science Citation Index Expanded (SCI-EXPANDED), Scopus, TR DİZİN (ULAKBİM)
  • Sayfa Sayıları: ss.203-211
  • Hacettepe Üniversitesi Adresli: Evet

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.