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

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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Autores principales: Ronen Bar-Yoseph, Galit Tal, Elena Dumin, Moneera Hanna, Gur Mainzer, Merav Zucker-Toledano, George Shallufi, Mira Jahshan, Hanna Mandel, Lea Bentur
Formato: article
Lenguaje:EN
Publicado: MDPI AG 2021
Materias:
infantile-onset Pompe disease (IOPD)
enzyme replacement therapy (ERT)
cardiopulmonary exercise testing (CPET)
6 min walking test (6MWT)
oxygen uptake at the peak of exercise (peak VO<sub>2</sub>)
Medicine
R
Acceso en línea:https://doaj.org/article/5abd37d53d554ddeadf7b6ea2ac12500
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https://doaj.org/article/5abd37d53d554ddeadf7b6ea2ac12500

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