Swiv plis pase 5 ane nan yon gwoup pasyan ki gen emofili B ki te trete ak Fidanacogene Elaparvovec Adeno-Associated Virus Gene Therapy.
Pwen esansyèl Soti nan 63yèm Reyinyon Anyèl ASH

Swiv plis pase 5 ane nan yon gwoup pasyan ki gen emofili B ki te trete ak Fidanacogene Elaparvovec Adeno-Associated Virus Gene Therapy.

Ben J. Samelson-Jones, MD, PhD1, Spencer K. Sullivan, MD2, John EJ Rasko, BSc (Med), MBBS (Hons), PhD, MAICD, FFSc (RCPA), FRCPA, FRACP, FAHMS3*, Adan Giermasz, MD, PhD4, Lindsey A. George, MD1,5, Jonatan M. Ducore, MD, MPH6, Jerome M. Teitel, MD, FRCPC7, Catherine E. McGuinn, MD8, Amanda O'Brien9*, Ian Winburn, MBBS, PhD, MRCS10 *, Lynne M Smith, MBA9*, Amit Chhabra, MBBS, MPH11, ak Jeremy Rupon, MD9

1Lopital Timoun Philadelphia, Philadelphia, PA
2Mississippi Sant pou Medsin Avanse, Madison, MS
3Terapi selil ak molekilè, Royal Prince Alfred Hospital, SLHD, Ostrali
4Inivèsite Kalifòni Davis, Sacramento, CA
5Perelman School of Medicine nan University of Pennsylvania, Philadelphia, PA
6Sant Tretman Emofili, UC Davis, Sacramento, CA
7University of Toronto, St Michael's Hospital, Toronto, Kanada
8Columbia University, New York, NY
9Pfizer Inc, Collegeville, PA
10Pfizer Ltd, Tadworth, Surrey, Wayòm Ini
11Pfizer Inc, New York, NY

Kle Done Pwen

FIX Aktivite sou tan apre perfusion nan Fidanacogene Elaparvovec

Aktivite FIX (apati dat limit Desanm 2020) pou 15 patisipan yo nan faz 1/2 esè klinik fidanacogene elaparvovec ki te resevwa yon dòz 5e11 vg/kg. Aktivite FIX te detèmine lè l sèvi avèk laboratwa santral Actin FSL ki baze sou yon etap koagulasyon tès la. Mwayèn nivo aktivite FIX yo te rete nan seri severite emofili modere: 22.8%, ane 1 (n = 15); 25.4%, ane 2 (n = 14); 22.9%, ane 3 (n = 14); 24.9%, ane 4 (n = 9); ak 19.8%, ane 5 (n = 7).

ABR sou tan apre perfusion nan Fidanacogene Elaparvovec

ABR pou 14 nan 15 faz 1/2 patisipan yo ki te patisipe nan etid swivi alontèm. Se sèlman 9 ak 5 patisipan yo te rive nan pwen tan swivi 5 ane ak 6 ane, respektivman. Depi pre-vektè perfusion, ABR vle di te varye ant 0.1-0.9 pou chak ane nan kou a nan swivi, epi pa gen okenn patisipan ki te rekòmanse pwofilaktik FIX.

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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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