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. |
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| 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 |
| ISSN: | 1750-1172 |
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| DOI: | 10.1186/s13023-026-04282-w |
