Evaluation of disease burden and response to treatment in adults with type 1 gaucher disease using a validated disease severity scoring system (DS3)

被引：0
作者：
Neal J. Weinreb
David N. Finegold
Eleanor Feingold
Zhen Zeng
Barry E. Rosenbloom
Suma P. Shankar
Dominick Amato
机构：
[1] University Research Foundation for Lysosomal Storage Diseases Inc.,Childrens Hospital of Pittsburgh
[2] One Children’s Hospital Drive,undefined
[3] University of Pittsburgh,undefined
[4] Tower Cancer Research Foundation,undefined
[5] Emory University School of Medicine,undefined
[6] Mount Sinai Hospital,undefined
来源：
Orphanet Journal of Rare Diseases | / 10卷
关键词：
Gaucher disease; DS3; Severity Score; Genotype; Splenectomy; Gaucher disease therapy; Treatment responses; Health state transitions; Gaucher bone disease; Bone event-free survival;
D O I：
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