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Blood Advances|July 18, 2024
Blood biodistribution and vector shedding of valoctocogene roxaparvovec in people with severe hemophilia ASuresh Agarwal, Krystal Sandza, Kristin Obrochta Moss, et al.
Molecular Therapy. Methods & Clinical Development|January 17, 2022
Molecular analysis of AAV5-hFVIII-SQ vector-genome-processing kinetics in transduced mouse and nonhuman primate liversChoong-Ryoul Sihn, Britta Handyside, Su Liu, et al.
Annals of Clinical and Translational Neurology|December 10, 2019
Neurofilament light is a treatment-responsive biomarker in CLN2 diseaseYuanbin Ru, Carley Corado, Russell K Soon, et al.
Arthritis Research & Therapy|November 30, 2013
A phase Ib multiple ascending dose study evaluating safety, pharmacokinetics, and early clinical response of brodalumab, a human anti-IL-17R antibody, in methotrexate-resistant rheumatoid arthritisDavid A Martin, Melvin Churchill, Luis Flores-Suarez, et al.
Haemophilia : the Official Journal of the World Federation of Hemophilia|February 6, 2024
Long-term safety and efficacy outcomes of valoctocogene roxaparvovec gene transfer up to 6 years post-treatmentEmily Symington, Savita Rangarajan, Will Lester, et al.
Arthritis & Rheumatology (Hoboken, N.J.)|January 25, 2017
Brief Report: Pharmacodynamics, Safety, and Clinical Efficacy of AMG 811, a Human Anti-Interferon-γ Antibody, in Patients With Discoid Lupus ErythematosusVictoria P Werth, David Fiorentino, Barbara A Sullivan, et al.
Human Gene Therapy|June 4, 2025
Recombinant Adeno-Associated Virus Integration Profiles in Nonhuman Primates and Gene Therapy Participants after Treatment with Valoctocogene RoxaparvovecChris B Russell, Christian Vettermann, Suresh Agarwal, et al.
Nature Medicine|April 12, 2022
Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia ASylvia Fong, Bridget Yates, Choong-Ryoul Sihn, et al.
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