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...

Volledige beschrijving

Bewaard in:
Bibliografische gegevens
Gepubliceerd in:Orphanet J Rare Dis
Hoofdauteurs: Schiffmann, Raphael, Pastores, Gregory M, Lien, Yeong-Hau H, Castaneda, Victoria, Chang, Peter, Martin, Rick, Wijatyk, Anna
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: BioMed Central 2014
Onderwerpen:
Research
Online toegang: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
Tags: Voeg label toe
Geen labels, Wees de eerste die dit record labelt!

Gelijkaardige items