Clinical immunogenicity outcomes from GENEr8-1, a phase 3 study of valoctocogene roxaparvovec, an AAV5-vectored gene therapy for hemophilia A
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Published:2024-07
Issue:7
Volume:32
Page:2052-2063
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ISSN:1525-0016
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Container-title:Molecular Therapy
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language:en
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Short-container-title:Molecular Therapy
Author:
Long Brian R.ORCID,
Robinson Tara M.,
Day Jonathan R.S.,
Yu Hua,
Lau Kelly,
Imtiaz Urooj,
Patton Kathryn S.,
de Hart Greg,
Henshaw Joshua,
Agarwal Suresh,
Vettermann Christian,
Zoog Stephen J.,
Gupta Soumi
Funder
BioMarin Pharmaceutical
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3. Patient perspectives on novel treatments in haemophilia: a qualitative study;van Balen;Patient,2020
4. Patient and parent preferences for characteristics of prophylactic treatment in hemophilia;Furlan;Patient Prefer. Adherence,2015
5. Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant;McIntosh;Blood,2013
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