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AMT-060 Terapi jèn nan adilt ki gen emofili grav oswa modere-grav B konfime ki eksprime ekspresyon fiks ak rediksyon dirab nan senyen ak faktè IX konsomasyon pou jiska 5 an
Pwen esansyèl nan 14yèm Kongrè Anyèl EAHAD la

AMT-060 Terapi jèn nan adilt ki gen emofili grav oswa modere-grav B konfime ki eksprime ekspresyon fiks ak rediksyon dirab nan senyen ak faktè IX konsomasyon pou jiska 5 an

Frank WG Leebeek, MD, PhD1, Karina Meijer, MD, PhD2, Michael Coppens, MD, PhD3, Pyè Kampmann, MD4, Robert Klamroth, MD5, Roger Schutgens, MD, PhD, MSc6, Giancarlo Castaman, MD7, Erhard Seifried, MD, PhD8, Joachim Schwaeble, MD9, Halvard Bönig, MD1,10, Eileen K. Sawyer, PhD11, ak Wolfgang Miesbach, MD, PhD12

1Sant Medikal Inivèsite Erasmus, Rotterdam, Netherlands

2Inivèsite Medikal Sant Groningen, Groningen, Netherlands

3Sant Medikal Amstèdam Inivèsite, Amstèdam, Netherlands

4Rigshospitalet, vil Copenhagen, Denmark

5Vivantes Klinikum, Bèlen, Almay

6Van Creveldkliniek, Inivèsite Sant Medikal Utrecht, Utrecht University, Utrecht, Netherlands

7Azienda Ospedaliera Università Careggi, Florence, Itali

8Enstiti Frankfurt, Alman Sèvis Kwa Wouj san Baden Wurtemberg-Hessen, Frankfurt, Almay

9Enstiti Frankfurt, Alman Sèvis Kwa Wouj san Baden Wurtemberg-Hessen, Frankfurt, Almay

10Inivèsite Washington, Seattle, WA

11uniQure Inc., Lexington, MA

12Inivèsite lopital Frankfurt, Frankfurt, Almay

Kle Done Pwen

FIX Aktivite soutni plis pase 4.5-5 ane nan 2 kòwòt dòz
AMT-060 te administre nan dòz 5x1012 gc / kg (kòwòt 1, N = 5) ak 2x1013 gc / kg (kòwòt 2, n = 5). Pandan 5 ane (kòwòt 1) ak 4.5 ane (kòwòt 2) nan swivi, vle di andojèn aktivite FIX te 5.2% (95% CI 2.0-8.4) nan kòwòt 1 ak 7.4% (95% CI 4.2-10.6) nan kòwòt 2. FIX aktivite te dòz depandan ak ki estab pandan peryòd la swivi pou tou de kòwòt. Valè nan parantèz reprezante aktivite FIX vle di sou tan. Se sèlman valè omwen 10 jou apre dènye administrasyon FIX konsantre yo enkli.
Rediksyon soutni nan senyen ak estime itilizasyon FIX

* Pwofilaksi te kon epi sispann pa 12 semèn si aktivite FIX te kenbe nan ≥ 2%; * Kowòt 2 done pou ane 5 reprezante 6 mwa. FIX, faktè IX

Nan tou de kòwòt dòz, vle di terapi ranplasman ABR ak FIX tonbe sevè pandan premye ane a apre perfusion e li te rete ba pou ane ki vin apre yo. Nan kòwòt 1, FIX l 'te redwi pa 84% ak ​​ABR pa 55% nan 5 ane relatif nan nivo pretreatment. Pou kòwòt 2, FIX l 'ak ABR te tou de redwi pa ~ 100% nan 4.5 ane 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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