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 Pastores; Milan Petakov; M. Judith Peterschmitt; Hanna Rosenbaum; Barry Rosenbloom; Lisa H. Underhill; Timothy M. Cox

doi:10.1016/j.bcmd.2019.04.003

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

Pediatrics

Research output: Contribution to journal › Letter › peer-review

2 Scopus citations

Original language	English (US)
Pages (from-to)	101-102
Number of pages	2
Journal	Blood Cells, Molecules, and Diseases
Volume	77
DOIs	https://doi.org/10.1016/j.bcmd.2019.04.003
State	Published - 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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10.1016/j.bcmd.2019.04.003

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Mistry, P. K., Balwani, M., Baris, H. N., Turkia, H. B., Burrow, T. A., Charrow, J., Cox, G. F., Danda, S., Dragosky, M., Drelichman, G., El-Beshlawy, A., Fraga, C., Freisens, S., Gaemers, S., Hadjiev, E., Kishnani, P. S., Lukina, E., Maison-Blanche, P., Martins, A. M., ... Cox, T. M. (2019). Addendum to Letter to the Editor: Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1. Blood Cells, Molecules, and Diseases, 77, 101-102. https://doi.org/10.1016/j.bcmd.2019.04.003

@article{9b962eea0b1547e083fa16b07021fc98,

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

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

note = "Funding Information: 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. Funding Information: 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 . ",

year = "2019",

month = jul,

doi = "10.1016/j.bcmd.2019.04.003",

language = "English (US)",

volume = "77",

pages = "101--102",

journal = "Blood Cells, Molecules, and Diseases",

issn = "1079-9796",

publisher = "Academic Press Inc.",

}

Mistry, PK, Balwani, M, Baris, HN, Turkia, HB, Burrow, TA, Charrow, J, Cox, GF, Danda, S, Dragosky, M, Drelichman, G, El-Beshlawy, A, Fraga, C, Freisens, S, Gaemers, S, Hadjiev, E, Kishnani, PS, Lukina, E, Maison-Blanche, P, Martins, AM, Pastores, G, Petakov, M, Peterschmitt, MJ, Rosenbaum, H, Rosenbloom, B, Underhill, LH & Cox, TM 2019, 'Addendum to Letter to the Editor: Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1', Blood Cells, Molecules, and Diseases, vol. 77, pp. 101-102. https://doi.org/10.1016/j.bcmd.2019.04.003

TY - JOUR

T1 - Addendum to Letter to the Editor

T2 - Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1

AU - Mistry, Pramod K.

AU - Balwani, Manisha

AU - Baris, Hagit N.

AU - Turkia, Hadhami Ben

AU - Burrow, T. Andrew

AU - Charrow, Joel

AU - Cox, Gerald F.

AU - Danda, Sumita

AU - Dragosky, Marta

AU - Drelichman, Guillermo

AU - El-Beshlawy, Amal

AU - Fraga, Cristina

AU - Freisens, Selena

AU - Gaemers, Sebastiaan

AU - Hadjiev, Evgueniy

AU - Kishnani, Priya S.

AU - Lukina, Elena

AU - Maison-Blanche, Pierre

AU - Martins, Ana Maria

AU - Pastores, Gregory

AU - Petakov, Milan

AU - Peterschmitt, M. Judith

AU - Rosenbaum, Hanna

AU - Rosenbloom, Barry

AU - Underhill, Lisa H.

AU - Cox, Timothy M.

N1 - Funding Information: 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. Funding Information: 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 .

PY - 2019/7

Y1 - 2019/7

UR - http://www.scopus.com/inward/record.url?scp=85064623158&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85064623158&partnerID=8YFLogxK

U2 - 10.1016/j.bcmd.2019.04.003

DO - 10.1016/j.bcmd.2019.04.003

M3 - Letter

C2 - 31029022

AN - SCOPUS:85064623158

SN - 1079-9796

VL - 77

SP - 101

EP - 102

JO - Blood Cells, Molecules, and Diseases

JF - Blood Cells, Molecules, and Diseases

ER -

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

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