Distal muscle weakness is a common and early feature in long-term enzyme-treated classic infantile Pompe patients

Distal muscle weakness is a common and early feature in long-term enzyme-treated classic infantile Pompe patients

Abstract Background Enzyme replacement therapy (ERT; alglucosidase alfa) has improved the prospects for patients with classic infantile Pompe disease considerably. However, over time we noticed that many of these children exhibit distal muscle weakness at an early age, which is in contrast to the pr...

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Bibliographic Details
Main Authors: J. J. A. van den Dorpel, E. Poelman, L. Harlaar, H. A. van Kooten, L. J. van der Giessen, P. A. van Doorn, A. T. van der Ploeg, J. M. P. van den Hout, N. A. M. E. van der Beek
Format: Article
Language:English
Published: BMC 2020-09-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Pompe disease
Glycogen storage disease type II
Enzyme replacement therapy (ERT)
Muscle function
Distal muscle weakness
Online Access:http://link.springer.com/article/10.1186/s13023-020-01482-w

Internet

http://link.springer.com/article/10.1186/s13023-020-01482-w

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