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Higher dosing of alglucosidase alfa improves outcomes in children with Pompe disease: a clinical study and review of the literature
PURPOSE: Enzyme replacement therapy (ERT) with recombinant human acid-α glucosidase (rhGAA) at standard dose of 20 mg/kg every other week is insufficient to halt the long-term progression of myopathy in Pompe disease. METHODS: We conducted a retrospective study on infantile-onset Pompe disease (IPD)...
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| Publicado no: | Genet Med |
|---|---|
| Main Authors: | , , , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2020
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7469631/ https://ncbi.nlm.nih.gov/pubmed/31904026 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41436-019-0738-0 |
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