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ba0002p181 | (1) | ICCBH2013

Long-term imiglucerase/alglucerase treatment in Latin American children with type 1 Gaucher disease: lessons from the International Collaborative Gaucher Group (ICGG) Gaucher Registry

Camelo Jr Jose Simon , Cabello Juan Francisco , Drelichman Guillermo G , Kerstenetzky Marcelo M , Sarmiento Isabel C , Linares Adriana

Objective: Evaluate the clinical characteristics of all Latin American pediatric patients with Gaucher disease type 1 (GD1) enrolled in the ICGG Gaucher Registry at baseline and investigate long-term outcomes and clinical benefit of prolonged imiglucerase/alglucerase therapy in patients with manifestations of GD1 at baseline.Methods: All Latin American patients with GD1 in the ICGG Gaucher Registry (NCT00358943) who were <18 years of age at the start...

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Long-term imiglucerase/alglucerase treatment in Latin American children with type 1 Gaucher disease: lessons from the International Collaborative Gaucher Group (ICGG) Gaucher Registry

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