Fehime Erdem Karapınar1, Havva Yazıcı1, Merve Yoldaş Çelik2

  • 1Pediatric division of inborn errors of metabolism, Ege Universitesi Tip Fakultesi, Izmir, Turkey.

Neuropediatrics
|December 26, 2025
PubMed
概括

早期酶替代疗法 (ERT) 显著减少了儿科庞佩病患者的临床事件. 综合护理对于改善这种罕见的溶酶体储存障碍的结果至关重要.

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