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Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), yon vektè amelyore pou transfè jèn nan granmoun ki gen emofil grav oswa modere-grav
Pwen esansyèl nan 14yèm Kongrè Anyèl EAHAD la

Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), yon vektè amelyore pou transfè jèn nan granmoun ki gen emofil grav oswa modere-grav: de ane done ki sòti nan yon faz 2b esè.

Annette von Drygalski, MD, PharmD1, Adan Giermasz, MD, PhD2, Giancarlo Castaman, MD3, Nigel S. Key, MBchB4, Susan U. Lattimore, RN5, Frank WG Leebeek, MD6, Wolfgang A. Miesbach, MD, PhD7, Michael Recht, MD, PhD5, Esteban Gomez, MD8, Robert Gut, MD, PhD9, ak Steven W. Pipe, MD10

1Inivèsite Kalifòni San Diego, La Jolla, CA, USA

2Inivèsite Kalifòni Davis, Sacramento, CA, USA

3Azienda Ospedaliera Università Careggi, Florence, Itali

4Inivèsite North Carolina, Chapel Hill, USA

5Oregon Sante ak Syans Inivèsite, Portland, USA

6Erasmus University Medical Center, Rotterdam, Netherlands

7Lopital Inivèsite Frankfurt, Almay

8Lopital Timoun Phoenix, Phoenix, USA

9uniQure Inc, Lexington, MA, Etazini

10Inivèsite Michigan, Ann Arbor, MI, USA

Kle Done Pwen

FIX Aktivite Apre AAV5-Padou hFIX vektè perfusion

Apre perfusion nan 2x1013 gc / kg, pwen final prensipal nan aktivite FIX ≥ 5% nan 6 semèn apre perfusion te reyalize nan tout 3 patisipan yo. Aktivite FIX vle di nan 2 zan te 44.2%, ak aktivite FIX te estab de ane 1 a ane 2.

Senyen Apre AAV5-Padova hFIX vektè perfusion

Plis pase 2 zan, 2 nan 3 patisipan yo pa te gen okenn senyen oswa itilize nan FIX konsantre. Patisipan nan twazyèm itilize yon total de 2 perfusion nan terapi ranplasman FIX (1 sispèk ak 1 konfime senyen) nan okazyon apa (eksepte operasyon). Tout patisipan yo rete pwofilaktik-gratis 2 zan apre perfusion.

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Sipòte pa sibvansyon edikasyon bay Bayer, BioMarin, CSL Behring, Freeline Therapeutics Limited, Pfizer Inc., Spark Therapeutics, ak uniQure, Inc.

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