Earlier and higher dosing of alglucosidase alfa improve outcomes in patients with infantile-onset Pompe disease: Evidence from real-world experiences

OBJECTIVE: Enzyme replacement therapy (ERT), the only approved therapy for infantile-onset Pompe disease (IOPD), had heterogeneous clinical effects due to factors such as severity, age at first treatment, dosage, and dosing regimens. We report the clinical and biochemical outcomes of a cohort of IOP...

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Bibliographische Detailangaben
Veröffentlicht in:Mol Genet Metab Rep
Hauptverfasser: Chien, Yin-Hsiu, Tsai, Wen-Hui, Chang, Chaw-Liang, Chiu, Pao-Chin, Chou, Yen-Yin, Tsai, Fuu-Jen, Wong, Siew-Lee, Lee, Ni-Chung, Hwu, Wuh-Liang
Format: Artigo
Sprache:Inglês
Veröffentlicht: Elsevier 2020
Schlagworte:
Research Paper
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7193123/
https://ncbi.nlm.nih.gov/pubmed/32373469
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2020.100591
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