作者
Stephen A Johnson, Ton Fang, Fabiola De Marchi, Dylan Neel, Donatienne Van Weehaeghe, James D Berry, Sabrina Paganoni
发表日期
2022/9
来源
Drugs
卷号
82
期号
13
页码范围
1367-1388
出版商
Springer International Publishing
简介
Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disorder involving loss of upper and lower motor neurons, with most cases ending in death within 3–5 years of onset. Several molecular and cellular pathways have been identified to cause ALS; however, treatments to stop or reverse disease progression are yet to be found. Riluzole, a neuroprotective agent offering only a modest survival benefit, has long been the sole disease-modifying therapy for ALS. Edaravone, which demonstrated statistically significant slowing of ALS disease progression, is gaining approval in an increasing number of countries since its first approval in 2015. Sodium phenylbutyrate and taurursodiol (PB-TURSO) was conditionally approved in Canada in 2022, having shown significant slowing of disease progression and prolonged survival. Most clinical trials have focused on testing small molecules affecting …
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