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Outcomes after 18 months of eliglustat therapy in treatment-naïve adults with Gaucher disease type 1: The phase 3 ENGAGE trial.
Mistry, Pramod K; Lukina, Elena; Ben Turkia, Hadhami; Shankar, Suma P; Baris, Hagit; Ghosn, Marwan; Mehta, Atul; Packman, Seymour; Pastores, Gregory; Petakov, Milan; Assouline, Sarit; Balwani, Manisha; Danda, Sumita; Hadjiev, Evgueniy; Ortega, Andres; Gaemers, Sebastiaan J M; Tayag, Regina; Peterschmitt, M Judith.
Afiliación
  • Mistry PK; Department of Internal Medicine and Pediatrics, Yale University School of Medicine, New Haven, CT, USA.
  • Lukina E; Department of Orphan Diseases, National Research Center for Hematology, Moscow, Russia.
  • Ben Turkia H; Department of Pediatrics, Hôpital La Rabta, Tunis, Tunisia.
  • Shankar SP; Department of Human Genetics, Emory University School of Medicine, Atlanta, GA, USA.
  • Baris H; Raphael Recanati Genetic Institute, Rabin Medical Center, Petach Tikvah, Israel.
  • Ghosn M; Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.
  • Mehta A; The Genetics Institute, Rambam Health Care Campus and The Ruth and Bruce Rappaport Faculty of Medicine, Technion - Israel Institute of Technology, Haifa, Israel.
  • Packman S; Department of Hematology-Oncology, Hôtel-Dieu de France University Hospital, Beirut, Lebanon.
  • Pastores G; Department of Haematology, The Royal Free Hospital, University College, London, UK.
  • Petakov M; Department of Pediatrics, UCSF School of Medicine, San Francisco, CA, USA.
  • Assouline S; Department of Neurology and Pediatrics, New York University School of Medicine, New York, NY, USA.
  • Balwani M; Department of Neuroendocrinology, Clinical Center of Serbia, Belgrade University Medical School, Serbia.
  • Danda S; Department of Medicine, Division of Hematology, Jewish General Hospital, Montreal, Quebec, Canada.
  • Hadjiev E; Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mt. Sinai Hospital, New York, NY, USA.
  • Ortega A; Department of Medical Genetics, Christian Medical College, Vellore, Tamil Nadu, India.
  • Gaemers SJM; Medical University - Sofia, Faculty of Medicine, Department of Internal diseases, UMHAT "Alexandrovska"- Clinic of Hematology, Sofia, Bulgaria.
  • Tayag R; OCA Hospital, Monterrey, Mexico.
  • Peterschmitt MJ; Global Pharmacovigilance, Sanofi Genzyme, Naarden, The Netherlands.
Am J Hematol ; 92(11): 1170-1176, 2017 Nov.
Article en En | MEDLINE | ID: mdl-28762527
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Pirrolidinas / Inhibidores Enzimáticos / Terapia de Reemplazo Enzimático / Enfermedad de Gaucher Tipo de estudio: Clinical_trials / Diagnostic_studies / Observational_studies / Prognostic_studies / Risk_factors_studies Límite: Humans Idioma: En Año: 2017 Tipo del documento: Article
Texto completo
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Pirrolidinas / Inhibidores Enzimáticos / Terapia de Reemplazo Enzimático / Enfermedad de Gaucher Tipo de estudio: Clinical_trials / Diagnostic_studies / Observational_studies / Prognostic_studies / Risk_factors_studies Límite: Humans Idioma: En Año: 2017 Tipo del documento: Article