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The Lancet. Haematology|November 9, 2024
Concizumab prophylaxis in people with haemophilia A or haemophilia B without inhibitors (explorer8): a prospective, multicentre, open-label, randomised, phase 3a trialPratima Chowdary, Pantep Angchaisuksiri, Shashikant Apte, et al.The New England Journal of Medicine|July 11, 2017
Targeting of Antithrombin in Hemophilia A or B with RNAi TherapyK John Pasi, Savita Rangarajan, Pencho Georgiev, et al.Thrombosis and Haemostasis|May 6, 2020
A Novel, Enriched Population Pharmacokinetic Model for Recombinant Factor VIII-Fc Fusion Protein Concentrate in Hemophilia A PatientsLaura H Bukkems, Jessica M Heijdra, Mary Mathias, et al.Blood|June 23, 2016
Efficacy and safety of rVIII-SingleChain: results of a phase 1/3 multicenter clinical trial in severe hemophilia AJohnny Mahlangu, Kazimierz Kuliczkowski, Faraizah Abdul Karim, et al.Blood|November 15, 2013
Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia AJohnny Mahlangu, Jerry S Powell, Margaret V Ragni, et al.Cytotherapy|April 3, 2026
A first-in-human phase I clinical trial investigating the safety and efficacy of repeat dosing MSCTRAIL cell therapy in addition to chemotherapy and immunotherapy in patients with advanced lung cancerRebecca N Graham, Alice Davies, Monica L Mullin, et al.The New England Journal of Medicine|June 11, 2025
Sustained Clinical Benefit of AAV Gene Therapy in Severe Hemophilia BUlrike M Reiss, Andrew M Davidoff, Edward G D Tuddenham, et al.The New England Journal of Medicine|November 20, 2014
Long-term safety and efficacy of factor IX gene therapy in hemophilia BAmit C Nathwani, Ulreke M Reiss, Edward G D Tuddenham, et al.The New England Journal of Medicine|December 14, 2011
Adenovirus-associated virus vector-mediated gene transfer in hemophilia BAmit C Nathwani, Edward G D Tuddenham, Savita Rangarajan, et al.Journal of Clinical Medicine|September 28, 2023
Nebulized Recombinant Tissue Plasminogen Activator (rt-PA) for Acute COVID-19-Induced Respiratory Failure: An Exploratory Proof-of-Concept TrialPratima Chowdary, Banwari Agarwal, Maria Rita Peralta, et al.Pageof 13