Distal muscle weakness is a common and early feature in long-term enzyme-treated classic infantile Pompe patients | Orphanet Journal of Rare Diseases | Full Text

Distal muscle weakness is a common and early feature in long-term enzyme-treated classic infantile Pompe patients | Orphanet Journal of Rare Diseases | Full Text

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

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...

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 and N. A. M. E. van der Beek

Citation:Orphanet Journal of Rare Diseases 2020 15:247

Content type:Research

 

Published on: 14 September 2020

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