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Eliglustat, an investigational oral therapy for Gaucher disease type 1: Phase 2 trial results after 4 years of treatment.
Lukina, Elena; Watman, Nora; Dragosky, Marta; Pastores, Gregory M; Arreguin, Elsa Avila; Rosenbaum, Hanna; Zimran, Ari; Angell, Jennifer; Ross, Leorah; Puga, Ana Cristina; Peterschmitt, Judith M.
Afiliação
  • Lukina E; Hematology Research Center of Ministry of Healthcare of the Russian Federation, Novy Zykovsky proezd 4, 125167 Moscow, Russia. Electronic address: kira-l@mail.ru.
  • Watman N; Hospital Ramos Mejia, Urquiza 609, Buenos Aires, Argentina. Electronic address: npwatman@yahoo.com.ar.
  • Dragosky M; Instituto Argentino de Diagnostico y Tratamiento, Marcelo T. de Alvear 2346/2400, Buenos Aires, Argentina. Electronic address: marta@dragosky.com.ar.
  • Pastores GM; National Gaucher Disease Treatment Center: Yale Lysosomal Disease Center, 333 Cedar Street, 1080 LMP, New Haven, CT 06519, USA. Electronic address: gregory.pastores@yale.edu.
  • Arreguin EA; Instituto Mexicano del Seguro Social, Hospital de Especialidades, Seris y Zaachila S/N, Mexico City, Mexico. Electronic address: elsa.avila@yahoo.com.mx.
  • Rosenbaum H; Hematology Ambulatory Services, Rambam Medical Center, 8 Haaliya St, Haifa 31096, Israel. Electronic address: h_rosenbaum@rambam.health.gov.il.
  • Zimran A; Shaare Zedek Medical Center, Gaucher Unit Floor 5, POB 3235, One Bezek Road, Jerusalem 91031, Israel. Electronic address: azimran@gmail.com.
  • Angell J; Genzyme, a Sanofi company, 500 Kendall Street, Cambridge, MA, 02142, USA. Electronic address: Jennifer.Shannon@genzyme.com.
  • Ross L; Genzyme, a Sanofi company, 500 Kendall Street, Cambridge, MA, 02142, USA. Electronic address: Leorah.Ross@genzyme.com.
  • Puga AC; Genzyme, a Sanofi company, 500 Kendall Street, Cambridge, MA, 02142, USA. Electronic address: AnaCristina.Puga@genzyme.com.
  • Peterschmitt JM; Genzyme, a Sanofi company, 500 Kendall Street, Cambridge, MA, 02142, USA. Electronic address: Judith.Peterschmitt@genzyme.com.
Blood Cells Mol Dis ; 53(4): 274-6, 2014 Dec.
Article em En | MEDLINE | ID: mdl-24835462
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Pirrolidinas / Drogas em Investigação / Inibidores Enzimáticos / Doença de Gaucher Tipo de estudo: Observational_studies / Prognostic_studies Limite: Adolescent / Adult / Female / Humans / Male / Middle aged Idioma: En Revista: Blood Cells Mol Dis Assunto da revista: HEMATOLOGIA Ano de publicação: 2014 Tipo de documento: Article
Texto completo
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Pirrolidinas / Drogas em Investigação / Inibidores Enzimáticos / Doença de Gaucher Tipo de estudo: Observational_studies / Prognostic_studies Limite: Adolescent / Adult / Female / Humans / Male / Middle aged Idioma: En Revista: Blood Cells Mol Dis Assunto da revista: HEMATOLOGIA Ano de publicação: 2014 Tipo de documento: Article