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

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 virus (A...

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Detalles Bibliográficos
Autores principales:	Hannah, William B., Case, Laura E., Smith, Edward C., Walters, Crista, Bali, Deeksha, Kishnani, Priya S., Koeberl, Dwight D.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley & Sons, Inc. 2023
Materias:	Research Reports
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10494494/ https://www.ncbi.nlm.nih.gov/pubmed/37701327 http://dx.doi.org/10.1002/jmd2.12391

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