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. 2024 Jan 3;8(3):102308.
doi: 10.1016/j.rpth.2023.102308. eCollection 2024 Mar.

The Evolution of Hemophilia Therapeutics: An Illustrated Review

María G Español  1   2 , Jessica N Mistretta  1 , Michael D Tarantino  1   3 , Jonathan C Roberts  1   3
Affiliations

The Evolution of Hemophilia Therapeutics: An Illustrated Review

María G Español et al. Res Pract Thromb Haemost. .

Abstract

Hemophilia is a rare genetic bleeding disorder historically associated with high morbidity and mortality. Some individuals with hemophilia suffer associated chronic joint disease, chronic pain, and other physical and mental health challenges. In the last 50 years, a better understanding of the pathophysiology of the disease has resulted in extraordinary therapeutic advances leading to enhanced quality of life and increased life expectancy. We present an illustrated review of the evolution of hemophilia treatment from the development of non-factor therapies to gene therapy.

Keywords: Hemophilia; bleeding disorder; gene therapy; hemophilia treatment; illustrated review; treatment.

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Conflict of interest statement

Dr. Michael D. Tarantino reports consulting fees from Pfizer, Sanofi, Sobi-Swedish Orphan Biovirtum, Takeda, Amgen, INC, Octapharma, Genentech, BioMarin, UCB Biosciences, Dova Pharmaceuticals, Novo Nordisk, and payment honoraria from Sobi, Genentech, Amgen, Novartis, BioMarin, and Sanofi. Dr. Jonathan C. Roberts reports grants from Takeda and Genetech, consulting fees from CSL Behring, F. Hoffman-La Roche AG, Sanofi, HEMA Biologics, Novartis, Novo Nordisk, Pfizer, Takeda, and Genentech. Dr. María G. Español reports participation on Sevenfact Advisory Board from HEMA biologics and payment honoraria from Octapharma.

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