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Paper Details

Long-term hematological, visceral, and growth outcomes in children with Gaucher disease type 3 treated with imiglucerase in the International Collaborative Gaucher Group Gaucher Registry.
Molecular Genetics and Metabolism
29
2017
D409H, GBA, GBA mutations, GD, GD1, GD2, GD3, Gaucher, Gaucher disease, Gaucher disease type 3, L444P, anemia, central nervous system involvement, children, deficiency of lysosomal acid -glucosidase, growth failure, hematological and visceral disease, hemoglobin, hepatosplenomegaly, imiglucerase, patients, platelet, thrombocytopenia
Author NameAffiliation
Pramod K MistryYale University School of Medicine
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