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Agalsidase alfa in pediatric patients with Fabry disease: a 6.5-year open-label follow-up study
BACKGROUND: Signs and symptoms of the X-linked disorder, Fabry disease (FD), can occur early during childhood with heterogeneous clinical manifestations including potential cardiac and renal dysfunction. Several studies support the efficacy of the enzyme replacement therapy (ERT) agalsidase alfa, in...
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| Publicado no: | Orphanet J Rare Dis |
|---|---|
| Main Authors: | , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
BioMed Central
2014
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4260255/ https://ncbi.nlm.nih.gov/pubmed/25425121 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-014-0169-6 |
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