[Clinical and genetic characteristics of infantile-onset Pompe disease and long-term efficacy of enzyme replacement therapy].
Sun YM; Wang YJ; Wang HF; Wu LL; Tao YJ; Zhu JM; Gao LC; Xie CH · 2026 · Zhonghua er ke za zhi = Chinese journal of pediatrics
WASTE classifies this as Abandoned Hypothesis · AI classification, approximate
A hypothesis was tested and not supported — a dead end worth knowing about before you pursue it.
Abstract (excerpt)
Objective: To analyze the clinical and genetic characteristics of infantile-onset Pompe disease (IOPD) and evaluate the long-term efficacy of enzyme replacement therapy (ERT). Methods: A retrospective cohort study was conducted on 24 IOPD…
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Metadata source: Europe PMC · DOI 10.3760/cma.j.cn112140-20251031-00968