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Hemophilia: a revolution in a single injection?
17 avril 2026
By Ana Espino | Published on April 17, 2026 | 4 min readHemophilia is a rare genetic disorder characterized by a deficiency in coagulation factor VIII (hemophilia A) or IX (hemophilia B). This condition leads to recurrent bleeding episodes that can result in severe complications such as joint damage, chronic pain, and, in some cases, life-threatening hemorrhages. Beyond its clinical impact, hemoph
Hemophilia is a rare genetic disorder characterized by a deficiency in coagulation factor VIII (hemophilia A) or IX (hemophilia B). This condition leads to recurrent bleeding episodes that can result in severe complications such as joint damage, chronic pain, and, in some cases, life-threatening hemorrhages. Beyond its clinical impact, hemophilia significantly affects the quality of life of patients and their families.
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Scientific reference
Thornburg CD, et al. Clinical perspective: Advancing hemophilia treatment through gene therapy approaches. Mol Ther. 2025 Jun 4;33(6):2350-2362. doi: 10.1016/j.ymthe.2025.04.023. Epub 2025 Apr 21. PMID: 40263938; PMCID: PMC12172285.
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