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Addendum to Letter to the Editor: Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1.

Autor: 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, AR, USA., Charrow J; Northwestern University Feinberg School of Medicine, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, 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.

Publikováno v: Blood cells, molecules & diseases [Blood Cells Mol Dis] 2019 Jul; Vol. 77, pp. 101-102. Date of Electronic Publication: 2019 Apr 09.

Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1.

Autor: 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.

Publikováno v: Blood cells, molecules & diseases [Blood Cells Mol Dis] 2018 Jul; Vol. 71, pp. 71-74. Date of Electronic Publication: 2018 Apr 09.

Stability is maintained in adults with Gaucher disease type 1 switched from velaglucerase alfa to eliglustat or imiglucerase: A sub-analysis of the eliglustat ENCORE trial.

Autor: Pleat R; Sanofi Genzyme, Cambridge, MA, USA., Cox TM; Department of Medicine, University of Cambridge, Addenbrooke's Hospital, Cambridge, UK., Burrow TA; Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA., Giraldo P; Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), IISAragón, Zaragoza, Spain., Goker-Alpan O; Lysosomal Disorders Research and Treatment Unit, Center for Clinical Trials, O&O Alpan, LLC, Fairfax, VA, USA., Rosenbloom BE; Cedars-Sinai/Tower Hematology Oncology, Beverly Hills, CA, USA., Croal LR; Sanofi Genzyme, Cambridge, MA, USA., Underhill LH; Sanofi Genzyme, Cambridge, MA, USA., Gaemers SJ; Sanofi Genzyme, Cambridge, MA, USA., Peterschmitt MJ; Sanofi Genzyme, Cambridge, MA, USA.

Publikováno v: Molecular genetics and metabolism reports [Mol Genet Metab Rep] 2016 Sep 30; Vol. 9, pp. 25-28. Date of Electronic Publication: 2016 Sep 30 (Print Publication: 2016).