The global impact of imiglucerase therapy in children with Gaucher disease types 1 and 3: a real-world analysis from the International Collaborative Gaucher Group Gaucher Registry
伊米苷酶疗法对1型和3型戈谢病患儿的全球影响:来自国际戈谢病协作组戈谢病注册中心的真实世界分析
期刊:Orphanet Journal of Rare Diseases
影响因子:3.5
doi:10.1186/s13023-026-04282-w
Mistry, Pramod K; Carwile, Jenny L; Burrow, Thomas Andrew; Tantawy, Azza Abdel Gawad; Camelo, Jose Simon; Ganesh, Jaya; Batsu, Isabela; Perichon, Maria Gabriela; Oliveira-Dos-Santos, Antonio; Batista, Julie L