Long-term outcomes of very early treated infantile-onset Pompe disease with short-term steroid premedication: experiences from a nationwide newborn screening programme.

Chia-Feng Yang,Ting-Wei Ernie Liao,Yen-Ling Chu,Li-Zhen Chen,Ling-Yi Huang,Tsui-Feng Yang,Hui-Chen Ho,Shu-Min Kao, Dau-Ming Niu

Journal of medical genetics（2023）

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摘要

Very early ERT using our rapid diagnostic and treatment strategy enabled our patients with IOPD to have better outcomes than patients in other medical centres.

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Congenital, Hereditary, and Neonatal Diseases and Abnormalities,Disease Management,Early Diagnosis,Genetics, Medical,Pediatrics

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