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Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1.
Mistry, Pramod K; Balwani, Manisha; Baris, Hagit N; Turkia, Hadhami Ben; Burrow, T Andrew; Charrow, Joel; Cox, Gerald F; Danda, Sumita; Dragosky, Marta; Drelichman, Guillermo; El-Beshlawy, Amal; Fraga, Cristina; Freisens, Selena; Gaemers, Sebastiaan; Hadjiev, Evgueniy; Kishnani, Priya S; Lukina, Elena; Maison-Blanche, Pierre; Martins, Ana Maria; Pastores, Gregory; Petakov, Milan; Peterschmitt, M Judith; Rosenbaum, Hanna; Rosenbloom, Barry; Underhill, Lisa H; Cox, Timothy M.
Afiliação
  • Mistry PK; Yale University School of Medicine, New Haven, CT, USA.
  • Balwani M; Icahn School of Medicine at Mount Sinai, New York, NY, USA.
  • Baris HN; The Genetics Institute, Rambam Health Care Campus, The Ruth and Bruce Rappaport Faculty of Medicine, Technion, - Israel Institute of Technology, Haifa, Israel.
  • Turkia HB; Hôpital La Rabta, Tunis, Tunisia.
  • Burrow TA; College of Medicine, University of Arkansas for Medical Sciences, Little Rock, Arkansas, USA.
  • Charrow J; Northwestern University Feinberg School of Medicine, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.
  • Cox GF; Editas, Cambridge, MA, USA (formerly Sanofi Genzyme, Cambridge, MA, USA).
  • Danda S; Christian Medical College, Vellore, India.
  • Dragosky M; IMAI-Research, Buenos Aires, Argentina.
  • Drelichman G; Hospital de Niños Ricardo Gutiérrez, Buenos Aires, Argentina.
  • El-Beshlawy A; Pediatric Hematology, Cairo University, Egypt.
  • Fraga C; HDES Hospital, Ponta Delgada, Açores, Portugal.
  • Freisens S; Sanofi Genzyme, Cambridge, MA, USA.
  • Gaemers S; Sanofi Genzyme, Cambridge, MA, USA.
  • Hadjiev E; University Hospital Alexandrovska, Sofia, Bulgaria.
  • Kishnani PS; Duke University School of Medicine, Department of Pediatrics, Durham, NC, USA.
  • Lukina E; National Research Center for Hematology, Moscow, Russia.
  • Maison-Blanche P; Bichat University Hospital, Paris, France.
  • Martins AM; Universidade Federal de São Paulo, São Paulo, SP, Brazil.
  • Pastores G; New York University School of Medicine, New York, NY, USA.
  • Petakov M; Clinical Center of Serbia, University of Belgrade School of Medicine, Belgrade, Serbia.
  • Peterschmitt MJ; Sanofi Genzyme, Cambridge, MA, USA.
  • Rosenbaum H; Rambam Medical Center, Haifa, Israel.
  • Rosenbloom B; Cedars-Sinai, Tower Hematology Oncology, Beverly Hills, CA, USA.
  • Underhill LH; Sanofi Genzyme, Cambridge, MA, USA.
  • Cox TM; University of Cambridge, Department of Medicine, Box 157, Level 5, Addenbrooke's Hospital, Cambridge CB2 0QQ, UK. Electronic address: tmc12@medschl.cam.ac.uk.
Blood Cells Mol Dis ; 71: 71-74, 2018 07.
Article em En | MEDLINE | ID: mdl-29680197
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doença de Gaucher Limite: Humans Idioma: En Ano de publicação: 2018 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doença de Gaucher Limite: Humans Idioma: En Ano de publicação: 2018 Tipo de documento: Article