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 YangTing-Wei Ernie LiaoYen-Ling ChuLi-Zhen ChenLing-Yi HuangTsui-Feng YangHui-Chen HoShu-Min KaoDau-Ming NiuPublished in: Journal of medical genetics (2022)
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.