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.2018.04.001

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

20 Scopus citations

Original language	English (US)
Pages (from-to)	71-74
Number of pages	4
Journal	Blood Cells, Molecules, and Diseases
Volume	71
DOIs	https://doi.org/10.1016/j.bcmd.2018.04.001
State	Published - Jul 2018

ASJC Scopus subject areas

Molecular Medicine
Molecular Biology
Hematology
Cell Biology

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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. (2018). Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1. Blood Cells, Molecules, and Diseases, 71, 71-74. https://doi.org/10.1016/j.bcmd.2018.04.001

@article{3ec86359606440729db9806338a64a22,

title = "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.}",

year = "2018",

month = jul,

doi = "10.1016/j.bcmd.2018.04.001",

language = "English (US)",

volume = "71",

pages = "71--74",

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

issn = "1079-9796",

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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 2018, 'Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1', Blood Cells, Molecules, and Diseases, vol. 71, pp. 71-74. https://doi.org/10.1016/j.bcmd.2018.04.001

TY - JOUR

T1 - 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.

PY - 2018/7

Y1 - 2018/7

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UR - http://www.scopus.com/inward/citedby.url?scp=85046818875&partnerID=8YFLogxK

U2 - 10.1016/j.bcmd.2018.04.001

DO - 10.1016/j.bcmd.2018.04.001

M3 - Letter

C2 - 29680197

AN - SCOPUS:85046818875

SN - 1079-9796

VL - 71

SP - 71

EP - 74

JO - Blood Cells, Molecules, and Diseases

JF - Blood Cells, Molecules, and Diseases

ER -

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

ASJC Scopus subject areas

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