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...

Полное описание

Сохранить в:
Библиографические подробности
Опубликовано в: :Orphanet J Rare Dis
Главные авторы: Schiffmann, Raphael, Pastores, Gregory M, Lien, Yeong-Hau H, Castaneda, Victoria, Chang, Peter, Martin, Rick, Wijatyk, Anna
Формат: Artigo
Язык:Inglês
Опубликовано: BioMed Central 2014
Предметы:
Research
Online-ссылка: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
Метки: Добавить метку
Нет меток, Требуется 1-ая метка записи!

Схожие документы