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

Pramod K. Mistry, Manisha Balwani, Hagit N. Baris, Hadhami Ben Turkia, T. Andrew Burrow, Joel Charrow, Gerald F. Cox, Sumita Danda, Marta Dragosky, Guillermo Drelichman, Amal El-Beshlawy, Cristina Fraga, Selena Freisens, Sebastiaan Gaemers, Evgueniy Hadjiev, Priya S. Kishnani, Elena Lukina, Pierre Maison-Blanche, Ana Maria Martins, Gregory PastoresMilan Petakov, M. Judith Peterschmitt, Hanna Rosenbaum, Barry Rosenbloom, Lisa H. Underhill, Timothy M. Cox*

*Corresponding author for this work

Research output: Contribution to journalLetterpeer-review

2 Scopus citations
Original languageEnglish (US)
Pages (from-to)101-102
Number of pages2
JournalBlood Cells, Molecules, and Diseases
Volume77
DOIs
StatePublished - Jul 2019

Funding

Pramod Mistry: Lead principle investigator in the eliglustat ENGAGE trial and principal investigator in the eliglustat ENCORE trial. Member of the International Collaborative Gaucher Group (ICCG) Gaucher Registry North American Advisory Board. Receives research support from Sanofi Genzyme and honoraria and travel reimbursement from Sanofi Genzyme.Hagit N. Baris: Principal investigator in the eliglustat ENGAGE trial. Recipient of research grants from Pfizer, Sanofi Genzyme, Shire, of honoraria from Sanofi Genzyme and Shire, of travel grants from Genzyme, Shire, Protalix and Pfizer and advisory board member of Sanofi Genzyme and Shire.Amal El Beshlawy: Principal investigator in the eliglustat ENCORE trial. Has received a research grant from Sanofi Genzyme.Priya Kishnani: Principal investigator in the eliglustat ENCORE trial. Received research/grant support from Sanofi Genzyme, Shire Pharmaceuticals, and Pfizer; and has received consulting fees and honoraria from Sanofi Genzyme, Shire Pharmaceuticals. Member of the ICGG Gaucher Registry Advisory Board for Sanofi Genzyme; Scientific Advisory Board for Shire Pharmaceuticals.Gregory Pastores: Principal investigator in the eliglustat ENGAGE and ENCORE trials. Has received grant/research support from Actelion, Alexion, Amicus/GSK, BioMarin, Sanofi Genzyme, Protalix BioTherapeutics/Pfizer, and Shire.Hanna Rosenbaum: Principal investigator in the eliglustat Phase 2 trial. Has received honoraria and travel reimbursement from Sanofi Genzyme. Recipient of research grants from Pfizer and Sanofi Genzyme.Timothy M. Cox: Lead principal investigator in the eliglustat ENCORE trial. Has received Honoraria, travel reimbursement and grant/research support from Shire Pharmaceuticals and an honorarium from Amicus/GSK. Hanna Rosenbaum: Principal investigator in the eliglustat Phase 2 trial. Has received honoraria and travel reimbursement from Sanofi Genzyme. Recipient of research grants from Pfizer and Sanofi Genzyme .

ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology
  • Hematology
  • Cell Biology

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