Recent therapeutic developments in spinal muscular atrophy

BORA, GAMZE; YEŞBEK KAYMAZ, AYŞE; Bekircan Kurt, Can; HALİLOĞLU, VİLDAN; TOPALOĞLU, HALUK; Erdem Yurter, HAYAT; ERDEM ÖZDAMAR, SEVİM

doi:10.3906/sag-1712-1

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

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
Açık Arşiv Koleksiyonu: AVESİS Açık Erişim Koleksiyonu
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.