Etranacogene dezaparvovec (AMT-061 phase 2b): Normal/near normal FIX activity and bleed cessation in hemophilia B

Annette Von Drygalski; Adam Giermasz; Giancarlo Castaman; Nigel S. Key; Susan Lattimore; Frank W.G. Leebeek; Wolfgang Miesbach; Michael Recht; Alison Long; Robert Gut; Eileen K. Sawyer; Steven W. Pipe

doi:10.1182/bloodadvances.2019000811

Etranacogene dezaparvovec (AMT-061 phase 2b): Normal/near normal FIX activity and bleed cessation in hemophilia B

Annette Von Drygalski, Adam Giermasz, Giancarlo Castaman, Nigel S. Key, Susan Lattimore, Frank W.G. Leebeek, Wolfgang Miesbach, Michael Recht, Alison Long, Robert Gut, Eileen K. Sawyer, Steven W. Pipe

Pediatrics

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

75 Scopus citations

Original language	English (US)
Pages (from-to)	3241-3247
Number of pages	7
Journal	Blood Advances
Volume	3
Issue number	21
DOIs	https://doi.org/10.1182/bloodadvances.2019000811
State	Published - Nov 12 2019

ASJC Scopus subject areas

Hematology

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Drygalski, A. V., Giermasz, A., Castaman, G., Key, N. S., Lattimore, S., Leebeek, F. W. G., Miesbach, W., Recht, M., Long, A., Gut, R., Sawyer, E. K., & Pipe, S. W. (2019). Etranacogene dezaparvovec (AMT-061 phase 2b): Normal/near normal FIX activity and bleed cessation in hemophilia B. Blood Advances, 3(21), 3241-3247. https://doi.org/10.1182/bloodadvances.2019000811

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author = "Drygalski, {Annette Von} and Adam Giermasz and Giancarlo Castaman and Key, {Nigel S.} and Susan Lattimore and Leebeek, {Frank W.G.} and Wolfgang Miesbach and Michael Recht and Alison Long and Robert Gut and Sawyer, {Eileen K.} and Pipe, {Steven W.}",

note = "Funding Information: This study was supported by research funding from uniQure B.V. Michael Lappin, a medical writer supported by funding from uniQure, provided drafts and editorial assistance to the authors during preparation of the manuscript.",

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doi = "10.1182/bloodadvances.2019000811",

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AU - Giermasz, Adam

AU - Castaman, Giancarlo

AU - Key, Nigel S.

AU - Lattimore, Susan

AU - Leebeek, Frank W.G.

AU - Miesbach, Wolfgang

AU - Recht, Michael

AU - Long, Alison

AU - Gut, Robert

AU - Sawyer, Eileen K.

AU - Pipe, Steven W.

N1 - Funding Information: This study was supported by research funding from uniQure B.V. Michael Lappin, a medical writer supported by funding from uniQure, provided drafts and editorial assistance to the authors during preparation of the manuscript.

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Etranacogene dezaparvovec (AMT-061 phase 2b): Normal/near normal FIX activity and bleed cessation in hemophilia B

ASJC Scopus subject areas

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