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

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 primarily...

Täydet tiedot

Tallennettuna:
Bibliografiset tiedot
Julkaisussa:Orphanet J Rare Dis
Päätekijät: van den Dorpel, J. J. A., Poelman, E., Harlaar, L., van Kooten, H. A., van der Giessen, L. J., van Doorn, P. A., van der Ploeg, A. T., van den Hout, J. M. P., van der Beek, N. A. M. E.
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: BioMed Central 2020
Aiheet:
Research
Linkit:https://ncbi.nlm.nih.gov/pmc/articles/PMC7488760/
https://ncbi.nlm.nih.gov/pubmed/32928284
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-020-01482-w
Tagit: Lisää tagi
Ei tageja, Lisää ensimmäinen tagi!

Samankaltaisia teoksia