医学
疾病
临床试验
脊髓小脑共济失调
神经刺激
心理干预
重症监护医学
生物信息学
神经科学
精神科
心理学
内科学
生物
刺激
作者
Shaila Ghanekar,Sheng‐Han Kuo,Joseph Staffetti,Theresa Zesiewicz
标识
DOI:10.1080/14737175.2022.2029703
摘要
Spinocerebellar ataxias (SCA) are a group of rare neurodegenerative diseases that dramatically affect the lives of affected individuals and their families. Despite having a clear understanding of SCA's etiology, there are no current symptomatic or neuroprotective treatments approved by the FDA.Research efforts have greatly expanded the possibilities for potential treatments, including both pharmacological and non-pharmacological interventions. Great attention is also being given to novel therapeutics based in gene therapy, neurostimulation, and molecular targeting. This review article will address the current advances in the treatment of SCA and what potential interventions are on the horizon.SCA is a highly complex and multifaceted disease family with the majority of research emphasizing symptomatic pharmacologic therapies. As pre-clinical trials for SCA and clinical trials for other neurodegenerative conditions illuminate the efficacy of disease modifying therapies such as AAV-mediated gene therapy and ASOs, the potential for addressing SCA at the pre-symptomatic stage is increasingly promising.
科研通智能强力驱动
Strongly Powered by AbleSci AI