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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...
Uloženo v:
| Vydáno v: | J Inherit Metab Dis |
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| Hlavní autoři: | , , , , , |
| Médium: | Artigo |
| Jazyk: | Inglês |
| Vydáno: |
Springer Netherlands
2018
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| Témata: | |
| On-line přístup: | 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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