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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.

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Title: 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.
Authors: Mistry PK; Yale University School of Medicine, 333 Cedar Street, LMP 1080, New Haven, CT, 06520, USA. Pramod.mistry@yale.edu., Carwile JL; Sanofi, Cambridge, MA, USA., Burrow TA; Arkansas Children's Hospital, University of Arkansas for Medical Sciences, Little Rock, AR, USA., Tantawy AAG; Ain Shams University, Cairo, Egypt., Camelo JS; Ribeirão Preto Medical School, University of São Paulo, São Paulo, Brazil., Ganesh J; Icahn School of Medicine at Mount Sinai, New York, NY, USA., Batsu I; Sanofi, Bridgewater, NJ, USA., Perichon MG; Sanofi, Cambridge, MA, USA., Oliveira-Dos-Santos A; Sanofi, Rio de Janeiro, Brazil., Batista JL; Sanofi, Cambridge, MA, USA.
Source: Orphanet journal of rare diseases [Orphanet J Rare Dis] 2026 Mar 11; Vol. 21 (1). Date of Electronic Publication: 2026 Mar 11.
Publication Type: Journal Article
Journal Info: Publisher: BioMed Central Country of Publication: England NLM ID: 101266602 Publication Model: Electronic Cited Medium: Internet ISSN: 1750-1172 (Electronic) Linking ISSN: 17501172 NLM ISO Abbreviation: Orphanet J Rare Dis Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: 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.
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  Data: <searchLink fieldCode="AU" term="%22Mistry+PK%22">Mistry PK</searchLink>; Yale University School of Medicine, 333 Cedar Street, LMP 1080, New Haven, CT, 06520, USA. Pramod.mistry@yale.edu.<br /><searchLink fieldCode="AU" term="%22Carwile+JL%22">Carwile JL</searchLink>; Sanofi, Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Burrow+TA%22">Burrow TA</searchLink>; Arkansas Children's Hospital, University of Arkansas for Medical Sciences, Little Rock, AR, USA.<br /><searchLink fieldCode="AU" term="%22Tantawy+AAG%22">Tantawy AAG</searchLink>; Ain Shams University, Cairo, Egypt.<br /><searchLink fieldCode="AU" term="%22Camelo+JS%22">Camelo JS</searchLink>; Ribeirão Preto Medical School, University of São Paulo, São Paulo, Brazil.<br /><searchLink fieldCode="AU" term="%22Ganesh+J%22">Ganesh J</searchLink>; Icahn School of Medicine at Mount Sinai, New York, NY, USA.<br /><searchLink fieldCode="AU" term="%22Batsu+I%22">Batsu I</searchLink>; Sanofi, Bridgewater, NJ, USA.<br /><searchLink fieldCode="AU" term="%22Perichon+MG%22">Perichon MG</searchLink>; Sanofi, Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Oliveira-Dos-Santos+A%22">Oliveira-Dos-Santos A</searchLink>; Sanofi, Rio de Janeiro, Brazil.<br /><searchLink fieldCode="AU" term="%22Batista+JL%22">Batista JL</searchLink>; Sanofi, Cambridge, MA, USA.
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        Value: 10.1186/s13023-026-04282-w
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      – TitleFull: 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.
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            – D: 11
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              Text: 2026 Mar 11
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