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Paper Title
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.
PubMed
Paper Journal Title
Molecular Genetics and Metabolism
Paper Citation Count
29
Paper Publication Year
2017
Bio Mention
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
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Pramod K Mistry
Yale University School of Medicine
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