Recent therapeutic developments in spinal muscular atrophy

Autor: Bora G, Ayşe YK, Can Ebru BK, Vildan Göknur H, Haluk Aydın T, Hayat EY, Sevim EÖ
Jazyk: angličtina
Zdroj: Turkish journal of medical sciences [Turk J Med Sci] 2018 Apr 30; Vol. 48 (2), pp. 203-211. Date of Electronic Publication: 2018 Apr 30.
DOI: 10.3906/sag-1712-1
Abstrakt: 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.
Databáze: MEDLINE