Survival and developmental milestones among Pompe registry patients with classic infantile-onset Pompe disease with different timing of initiation of treatment with enzyme replacement therapy (ERT)

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• 作者：Yin-Hsiu Chien ; Ans van der Ploeg ; Simon A. Jones ; Barry Byrne ; Ashok Vellodi ; Nancy Leslie ; Eugen Mengel ; Suma Shankar ; Pranoot Tanpaiboon ; David W. Stockton ; Julia B. Hennermann ; Zsuzsanna Devecseri ; Judy Kempf ; Joan Keutzer ; Priya S. Kishnani
• 刊名：Molecular Genetics and Metabolism
• 出版年：February, 2014
• 年：2014
• 卷：111
• 期：2
• 页码：S31-S32
• 全文大小：68 K

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