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Gaucher disease type 1 patients from the ICGG Gaucher Registry sustain initial clinical improvements during twenty years of imiglucerase treatment.
Weinreb, Neal J; Camelo, José Simon; Charrow, Joel; McClain, Monica R; Mistry, Pramod; Belmatoug, Nadia.
  • Weinreb NJ; Departments of Human Genetics and Medicine (Hematology), University of Miami Miller School of Medicine, Miami, FL, USA. Electronic address: N.weinreb@med.miami.edu.
  • Camelo JS; Department of Pediatrics, Ribeirão Preto Medical School, University of São Paulo, Bandeirantes Av., 3900 - 5th floor - Off D506 - HC Criança, São Paulo, Brazil. Electronic address: jscamelo@fmrp.usp.br.
  • Charrow J; Northwestern University Feinberg School of Medicine, 225 E Chicago Ave, Chicago, IL 60611, USA. Electronic address: jcharrow@northwestern.edu.
  • McClain MR; Sanofi Genzyme, 50 Binney Street, Cambridge, MA 02142, USA. Electronic address: mmcclain@acmg.net.
  • Mistry P; Department of Internal Medicine, Yale University School of Medicine, The Anlyan Center Building Room S217B, 300 Cedar Street, New Haven, CT 06520, USA. Electronic address: pramod.mistry@yale.edu.
  • Belmatoug N; Department of Internal Medicine, Referral Center for Lysosomal Diseases Assistance Publique-Hôpitaux de Paris, Paris Université, Beaujon Hospital, 100 Boulevard du Général Leclerc, 92110 Clichy, France. Electronic address: nadia.belmatoug@aphp.fr.
Mol Genet Metab ; 132(2): 100-111, 2021 02.
Article en En | MEDLINE | ID: mdl-33485799
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Terapia de Reemplazo Enzimático / Enfermedad de Gaucher / Glucosilceramidasa Límite: Adolescent / Adult / Child / Child, preschool / Female / Humans / Infant / Male / Middle aged Idioma: En Año: 2021 Tipo del documento: Article
Texto completo
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Terapia de Reemplazo Enzimático / Enfermedad de Gaucher / Glucosilceramidasa Límite: Adolescent / Adult / Child / Child, preschool / Female / Humans / Infant / Male / Middle aged Idioma: En Año: 2021 Tipo del documento: Article