Screening data from 19 patients with late‐onset Pompe disease for a phase I clinical trial of AAV8 vector‐mediated gene therapy

Abstract Late‐onset Pompe disease (LOPD) is a multisystem disorder with significant myopathy. The standard treatment is enzyme replacement therapy (ERT), a therapy that is lifesaving, yet with limitations. Clinical trials have emerged for other potential treatment options, including adeno‐associated...

全面介绍

书目详细资料

Principais autores:	William B. Hannah, Laura E. Case, Edward C. Smith, Crista Walters, Deeksha Bali, Priya S. Kishnani, Dwight D. Koeberl
格式:	Artigo
语言:	Inglês
出版:	Wiley 2023-09-01
丛编:	JIMD Reports
主题:	adeno‐associated virus gene therapy Pompe disease
在线阅读:	https://doi.org/10.1002/jmd2.12391
标签:	添加标签 没有标签, 成为第一个标记此记录!