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Therapeutic Advances in Hematology|June 29, 2026
Treatment preferences of people with hemophilia and caregivers: a plain language summaryHui Lu, Travis Gould, Carmine Colavecchia, et al.Blood Advances|December 6, 2024
Long-term safety and efficacy of fitusiran prophylaxis, and perioperative management, in people with hemophilia A or BSteven W Pipe, Toshko Lissitchkov, Pencho Georgiev, et al.Research and Practice in Thrombosis and Haemostasis|February 3, 2025
Adeno-associated virus-based gene therapy for hemophilia-addressing the gapsWolfgang Miesbach, Paul Batty, Pratima Chowdary, et al.Haemophilia : the Official Journal of the World Federation of Hemophilia|December 8, 2025
Patient and Caregiver Preferences for Long-Term Prophylactic Treatment of Bleeds in Haemophilia: A Discrete-Choice ExperimentHui Lu, Travis Gould, Carmine Colavecchia, et al.Haemophilia : the Official Journal of the World Federation of Hemophilia|November 20, 2024
UKHCDO gene therapy taskforce: Guidance for implementation of haemophilia gene therapy into routine clinical practice for adultsPratima Chowdary, Beatriz Duran, Paul Batty, et al.Journal of Inherited Metabolic Disease|December 15, 2021
Oral treatment for mucopolysaccharidosis VI: Outcomes of the first phase IIa study with odiparcilNathalie Guffon, Pratima Chowdary, Elisa Leão Teles, et al.The New England Journal of Medicine|January 31, 2023
Efanesoctocog Alfa Prophylaxis for Patients with Severe Hemophilia AAnnette von Drygalski, Pratima Chowdary, Roshni Kulkarni, et al.Blood Advances|March 15, 2022
Long-term efficacy and safety of subcutaneous concizumab prophylaxis in hemophilia A and hemophilia A/B with inhibitorsAmy D Shapiro, Pantep Angchaisuksiri, Jan Astermark, et al.Journal of Thrombosis and Haemostasis : JTH|February 15, 2021
Targeting of antithrombin in hemophilia A or B with investigational siRNA therapeutic fitusiran-Results of the phase 1 inhibitor cohortK John Pasi, Toshko Lissitchkov, Vasily Mamonov, et al.Blood|August 25, 2019
Subcutaneous concizumab prophylaxis in hemophilia A and hemophilia A/B with inhibitors: phase 2 trial resultsAmy D Shapiro, Pantep Angchaisuksiri, Jan Astermark, et al.Pageof 13