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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Detalles Bibliográficos
Autores principales: Schiffmann, Raphael, Pastores, Gregory M, Lien, Yeong-Hau H, Castaneda, Victoria, Chang, Peter, Martin, Rick, Wijatyk, Anna
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2014
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4260255/
https://www.ncbi.nlm.nih.gov/pubmed/25425121
http://dx.doi.org/10.1186/s13023-014-0169-6

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4260255/
https://www.ncbi.nlm.nih.gov/pubmed/25425121
http://dx.doi.org/10.1186/s13023-014-0169-6

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