An open-label clinical trial of agalsidase alfa enzyme replacement therapy in children with Fabry disease who are naïve to enzyme replacement therapy

BACKGROUND: Following a drug manufacturing process change, safety/efficacy of agalsidase alfa were evaluated in enzyme replacement therapy (ERT)-naïve children with Fabry disease. METHODS: In an open-label, multicenter, Phase II study (HGT-REP-084; Shire), 14 children aged ≥7 years received 0.2 mg/k...

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Опубликовано в: :Drug Des Devel Ther
Главные авторы: Goker-Alpan, Ozlem, Longo, Nicola, McDonald, Marie, Shankar, Suma P, Schiffmann, Raphael, Chang, Peter, Shen, Yinghua, Pano, Arian
Формат: Artigo
Язык:Inglês
Опубликовано: Dove Medical Press 2016
Предметы:
Original Research
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC4887054/
https://ncbi.nlm.nih.gov/pubmed/27307708
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2147/DDDT.S102761
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