作者
Abdelrahman Elshafay, Truong Hong Hieu, Mohamed Fahmy Doheim, Mahmoud Attia Mohamed Kassem, Mohammed Fathi ELdoadoa, Sarah Keturah Holloway, Heba Abo-Elghar, Kenji Hirayama, Nguyen Tien Huy
发表日期
2019/3/6
来源
CNS drugs
卷号
33
页码范围
239-250
出版商
Springer International Publishing
简介
Background
Spinal muscular atrophy (SMA) is a neuromuscular disorder classified into four types based on the age of onset of the disease. Early onset is correlated with a higher mortality rate, mainly due to respiratory complications. Valproic acid (VPA) is a histone deacetylase (HDAC) inhibitor that has shown positive results on SMA both in experimental and cohort studies.
Objectives
This systematic review and meta-analysis aimed to investigate the efficacy and safety of VPA in patients with SMA.
Methods
Eleven databases were systematically searched on 30 May 2017 for clinical trials that reported the efficacy and safety of VPA in SMA patients. The primary outcome was the efficacy of VPA in terms of gross motor function and expression of both full-length spinal motor neuron (SMN) gene (FL-SMN) and exon 7-lacking …
学术搜索中的文章
A Elshafay, TH Hieu, MF Doheim, MAM Kassem… - CNS drugs, 2019