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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Pubblicato in:Orphanet J Rare Dis
Autori principali: Schiffmann, Raphael, Pastores, Gregory M, Lien, Yeong-Hau H, Castaneda, Victoria, Chang, Peter, Martin, Rick, Wijatyk, Anna
Natura: Artigo
Lingua:Inglês
Pubblicazione: BioMed Central 2014
Soggetti:
Research
Accesso online: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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