Emerging drugs for Duchenne muscular dystrophy.-猫眼课题宝

DOI：

10.1517/14728214.2012.691965

发表时间：

2012-06

期刊：

Expert opinion on emerging drugs

影响因子：

3.4

通讯作者：

中科院分区：

医学3区

文献类型：

作者：

Malik V;Rodino-Klapac LR;Mendell JR

来源链接：

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3486431/?report=classic

Duchenne muscular dystrophy (DMD) is the most common, severe childhood form of muscular dystrophy. Treatment is limited to glucocorticoids that have the benefit of prolonging ambulation by approximately 2 years and preventing scoliosis. Finding a more satisfactory treatment should focus on maintaining long-term efficacy with a minimal side effect profile. Authors discuss different therapeutic strategies that have been used in pre-clinical and clinical settings. Multiple treatment approaches have emerged. Most attractive are molecular-based therapies that can express the missing dystrophin protein (exon skipping or mutation suppression) or a surrogate gene product (utrophin). Other approaches include increasing the strength of muscles (myostatin inhibitors), reducing muscle fibrosis, and decreasing oxidative stress. Additional targets include inhibiting NF-κB to reduce inflammation, or promoting skeletal muscle blood flow and muscle contractility using phosphodiesterase inhibitors or nitric oxide (NO) donors. The potential for each of these treatment strategies to enter clinical trials is a central theme of discussion. The review emphasizes that the goal of treatment should be to find a product at least as good as glucocorticoids with a lower side effect profile or with a significant glucocorticoid sparing effect.