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Enzyme replacement therapy for infantile‐onset Pompe disease
BACKGROUND: Infantile‐onset Pompe disease is a rare and progressive autosomal‐recessive disorder caused by a deficiency of the lysosomal enzyme acid alpha‐glucosidase (GAA). Current treatment involves enzyme replacement therapy (with recombinant human alglucosidase alfa) and symptomatic therapies (e...
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| Publicado en: | Cochrane Database Syst Rev |
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| Main Authors: | , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado: |
John Wiley & Sons, Ltd
2017
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| Acceso en liña: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6486223/ https://ncbi.nlm.nih.gov/pubmed/29155436 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/14651858.CD011539.pub2 |
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