Transformation in pretreatment manifestations of Gaucher disease type 1 during two decades of alglucerase/imiglucerase enzyme replacement therapy in the International Collaborative Gaucher Group (ICGG) Gaucher Registry.

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Title: Transformation in pretreatment manifestations of Gaucher disease type 1 during two decades of alglucerase/imiglucerase enzyme replacement therapy in the International Collaborative Gaucher Group (ICGG) Gaucher Registry.
Authors: Mistry PK; Department of Internal Medicine, Yale University School of Medicine, New Haven, Connecticut., Batista JL; Sanofi Genzyme, Cambridge, Massachusetts., Andersson HC; Hayward Genetics Center, Tulane University Medical School, New Orleans, Louisiana., Balwani M; Icahn School of Medicine at Mount Sinai, New York City, New York., Burrow TA; College of Medicine, University of Arkansas for Medical Sciences, Little Rock, Arkansas., Charrow J; Northwestern University Feinberg School of Medicine, Chicago, Illinois., Kaplan P; Children's Hospital of Philadelphia, Philadelphia, Pennsylvania., Khan A; The University of Calgary, Calgary, Alberta, Canada., Kishnani PS; Division of Medical Genetics, Duke University School of Medicine, Durham, North Carolina., Kolodny EH; New York University School of Medicine, New York, New York., Rosenbloom B; Cedars-Sinai/Tower Hematology Oncology, Beverly Hills, California., Scott CR; University of Washington, Seattle, Washington., Weinreb N; Departments of Human Genetics and Medicine (Hematology), University of Miami Miller School of Medicine, Miami, Florida.
Source: American journal of hematology [Am J Hematol] 2017 Sep; Vol. 92 (9), pp. 929-939. Date of Electronic Publication: 2017 Jul 07.
Publication Type: Clinical Trial; Journal Article; Multicenter Study
Journal Info: Publisher: Wiley-Blackwell Country of Publication: United States NLM ID: 7610369 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1096-8652 (Electronic) Linking ISSN: 03618609 NLM ISO Abbreviation: Am J Hematol Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:1096-8652
DOI:10.1002/ajh.24801