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Cardiology and Therapy|August 4, 2019
Estimating the Prevalence of Transthyretin Amyloid Cardiomyopathy in a Large In-Hospital Database in JapanIan Winburn, Tomonori Ishii, Takuma Sumikawa, et al.Orphanet Journal of Rare Diseases|August 3, 2023
The impact of bleeding event frequency on health-related quality of life and work productivity outcomes in a European cohort of adults with haemophilia A: insights from the CHESS II studyLisa Young, Yong Chen, José Alvir, et al.Drugs - Real World Outcomes|August 10, 2024
Real-World Amount of Clotting Factor Products and Non-Factor Products Dispensed and Annual Medical Expenditures for Japanese Patients with Haemophilia AMasato Bingo, Katsuyuki Fukutake, Kanae Togo, et al.Theranostics|February 7, 2024
Understanding AAV vector immunogenicity: from particle to patientBijay P Dhungel, Ian Winburn, Candida da Fonseca Pereira, et al.Journal of Blood Medicine|December 25, 2023
Real-World Amount of Clotting Factor Concentrates Dispensed and Annual Medical Expenditures for Japanese Patients with Hemophilia BKatsuyuki Fukutake, Kanae Togo, Linghua Xu, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|January 13, 2023
Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapyMartin Schulz, Daniel I Levy, Christos J Petropoulos, et al.Haemophilia : the Official Journal of the World Federation of Hemophilia|September 27, 2022
How to assess, detect, and manage joint involvement in the era of transformational therapies: Role of point-of-care ultrasoundMatteo Nicola Dario Di Minno, Carlo Martinoli, Gianluigi Pasta, et al.Molecular Therapy. Methods & Clinical Development|July 18, 2024
Global seroprevalence of neutralizing antibodies against adeno-associated virus serotypes used for human gene therapiesAmit Chhabra, George Bashirians, Christos J Petropoulos, et al.Haemophilia : the Official Journal of the World Federation of Hemophilia|April 19, 2022
Examining patient and professional perspectives in the UK for gene therapy in haemophiliaIone Woollacott, George Morgan, Pratima Chowdary, et al.The New England Journal of Medicine|April 16, 2025
Fidanacogene Elaparvovec for Hemophilia B - A Multiyear Follow-up StudyJohn E J Rasko, Benjamin J Samelson-Jones, Lindsey A George, et al.Pageof 1