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Maria Elisa Mancuso, Daniel Eriksson, Aletta Falk, Zalmai Hakimi, Piotr Wojciechowski, Marlena Wdowiak & Robert Klamroth. (2023) Efficacy of rFIXFc versus N9-GP Prophylaxis in Patients with Hemophilia B: Matching-Adjusted Indirect Comparison of B-LONG and PARADIGM 2 Trials. Journal of Blood Medicine 14, pages 427-434.
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Articles from other publishers (7)
Antal Zemplenyi, Jim Leonard, Michael J. DiStefano, Kelly E. Anderson, Garth C. Wright, Nicholas D. Mendola, Kavita Nair & R. Brett McQueen. (2023) Using Real-World Data to Inform Value-Based Contracts for Cell and Gene Therapies in Medicaid. PharmacoEconomics.
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Andrea Guala & Emilia Parodi. (2023) Il farmaco più costoso del mondo. Medico e Bambino 42:7, pages 415-416.
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Bertrand Jordan. (2023) Une thérapie très attendue… et un prix exorbitant !. médecine/sciences 39:2, pages 187-190.
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Simon Fletcher, Kathryn Jenner, Luke Pembroke, Michael Holland & Kate Khair. (2022) The experiences of people with haemophilia and their families of gene therapy in a clinical trial setting: regaining control, the Exigency study. Orphanet Journal of Rare Diseases 17:1.
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Kristen Corrao & Lynn Malec. (2022) Disease Eradication for Hemophilia: How and Who. The Hematologist 19:6.
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Pratima ChowdarySusan ShapiroMike MakrisGillian EvansSara BoyceKate TalksGerard DolanUlrike ReissMark PhillipsAnne RiddellMaria R. PeraltaMichelle QuayeDavid W. PatchEdward TuddenhamAllison DaneMarie WatisséeAlison LongAmit Nathwani. (2022) Phase 1–2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B. New England Journal of Medicine 387:3, pages 237-247.
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Courtney D. Thornburg. (2021) Etranacogene dezaparvovec for hemophilia B gene therapy. Therapeutic Advances in Rare Disease 2, pages 263300402110588.
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