Long-term follow-up of 17 patients with childhood Pompe disease treated with enzyme replacement therapy

OBJECTIVES: Pompe disease is a progressive metabolic myopathy for which enzyme replacement therapy (ERT) was approved in 2006. While various publications have examined the effects of ERT in classic-infantile patients and in adults, little has been published on ERT in children with non-classic presen...

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Detaylı Bibliyografya
Yayımlandı:J Inherit Metab Dis
Asıl Yazarlar: van der Meijden, Jan C., Kruijshaar, Michelle E., Harlaar, Laurike, Rizopoulos, Dimitris, van der Beek, Nadine A. M. E., van der Ploeg, Ans T.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Springer Netherlands 2018
Konular:
Original Article
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC6326992/
https://ncbi.nlm.nih.gov/pubmed/29556838
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s10545-018-0166-3
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