Valoctocogene roxaparvovec gene transfer in participants with HIV-Reference-Cited by-同舟云学术

Valoctocogene roxaparvovec gene transfer in participants with HIV

Published:2023-04-14 Issue:8 Volume:7 Page:1525-1530
ISSN:2473-9529
Container-title:Blood Advances
language:en
Short-container-title:

Author:

Ragni Margaret V.¹,Majerus Elaine²,Fong Sylvia³,Yates Bridget³,Scheeler Stephen³,Razon Lisa³,Yu Hua³,Reddy Divya B.³,Robinson Tara M.³

Affiliation:

1. 1Medicine and Clinical and Translational Science, Department of Medicine, Division Hematology/Oncology, University of Pittsburgh Medical Center, Hemophilia Center of Western Pennsylvania, Pittsburgh, PA

2. 2Department of Medicine, Washington University in St. Louis, St. Louis, MO

3. 3BioMarin Pharmaceutical Inc., Novato, CA

Publisher

American Society of Hematology

Subject

Hematology

Link

https://ashpublications.org/bloodadvances/article-pdf/7/8/1525/2045815/blooda_adv-2022-008948-main.pdf

Reference31 articles.

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2. Real-world rates of bleeding, factor VIII use, and quality of life in individuals with severe haemophilia A receiving prophylaxis in a prospective, noninterventional study;Kenet;J Clin Med,2021

3. Patients' and parents' satisfaction with, and preference for, haemophilia A treatments: a cross-sectional, multicentre, observational study;Park;Haemophilia,2021

4. Patient perspectives on novel treatments in haemophilia: a qualitative study;van Balen;Patient,2020

5. Gene therapy with BMN 270 results in therapeutic levels of FVIII in mice and primates and normalization of bleeding in hemophilic mice;Bunting;Mol Ther,2018

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