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Individualized Assessment of Exercise Capacity in Response to Acute and Long-Term Enzyme Replacement Therapy in Pediatric Pompe Disease

Background: Enzyme replacement therapy (ERT) with alglucosidase alfa improves the prospect of patients with infantile-onset Pompe disease (IOPD). However, a progressive decline has been reported. Objective quantification of the response to ERT when assessing newer strategies is warranted. Methods: T...

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Detalles Bibliográficos
Autores principales:	Bar-Yoseph, Ronen, Tal, Galit, Dumin, Elena, Hanna, Moneera, Mainzer, Gur, Zucker-Toledano, Merav, Shallufi, George, Jahshan, Mira, Mandel, Hanna, Bentur, Lea
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2021
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8625342/ https://www.ncbi.nlm.nih.gov/pubmed/34834457 http://dx.doi.org/10.3390/jpm11111105

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