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Long-term benefit of enzyme replacement therapy in Pompe disease: A 5-year prospective study.

Authors
  • Kuperus, Esther1
  • Kruijshaar, Michelle E1
  • Wens, Stephan C A1
  • de Vries, Juna M1
  • Favejee, Marein M1
  • van der Meijden, Jan C1
  • Rizopoulos, Dimitris1
  • Brusse, Esther1
  • van Doorn, Pieter A1
  • van der Ploeg, Ans T1
  • van der Beek, Nadine A M E2
  • 1 From the Erasmus MC University Medical Center (E.K., S.C.A.W., J.M.d.V., E.B., P.A.v.D., N.A.M.E.v.d.B.), Center for Lysosomal and Metabolic Diseases, Department of Neurology; Erasmus MC University Medical Center-Sophia Children's Hospital (M.E.K., J.C.v.d.M., A.T.v.d.P.), Center for Lysosomal and Metabolic Diseases, Department of Pediatrics; Erasmus MC University Medical Center (M.M.F.), Center for Lysosomal and Metabolic Diseases, Department of Rehabilitation Medicine and Physical Therapy; and Erasmus MC University Medical Center (D.R.), Department of Biostatistics, Rotterdam, the Netherlands. , (Netherlands)
  • 2 From the Erasmus MC University Medical Center (E.K., S.C.A.W., J.M.d.V., E.B., P.A.v.D., N.A.M.E.v.d.B.), Center for Lysosomal and Metabolic Diseases, Department of Neurology; Erasmus MC University Medical Center-Sophia Children's Hospital (M.E.K., J.C.v.d.M., A.T.v.d.P.), Center for Lysosomal and Metabolic Diseases, Department of Pediatrics; Erasmus MC University Medical Center (M.M.F.), Center for Lysosomal and Metabolic Diseases, Department of Rehabilitation Medicine and Physical Therapy; and Erasmus MC University Medical Center (D.R.), Department of Biostatistics, Rotterdam, the Netherlands. [email protected] , (Netherlands)
Type
Published Article
Journal
Neurology
Publisher
Ovid Technologies (Wolters Kluwer) - American Academy of Neurology
Publication Date
Nov 08, 2017
Identifiers
DOI: 10.1212/WNL.0000000000004711
PMID: 29117951
Source
Medline
License
Unknown

Abstract

This study provides Class IV evidence that for patients with Pompe disease, long-term ERT positively affects muscle strength, pulmonary function, and daily life activities.

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