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...

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Auteurs principaux:	William B. Hannah, Laura E. Case, Edward C. Smith, Crista Walters, Deeksha Bali, Priya S. Kishnani, Dwight D. Koeberl
Format:	Artigo
Langue:	Inglês
Publié:	Wiley 2023-09-01
Collection:	JIMD Reports
Sujets:	adeno‐associated virus gene therapy Pompe disease
Accès en ligne:	https://doi.org/10.1002/jmd2.12391
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