Physical activity (PA) is vital in child development, while inactivity remains a leading contributor to global mortality. Therapeutic advances have enabled greater PA in boys with haemophilia (BwH), yet...
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Gene therapy (GT) provides sustained FVIII levels without repeated infusions in Hemophilia A (HA) patients thus overcoming a major limitation of replacement therapy. However, issues remain e.g., patient...
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Advances in hemophilia treatment, including extended half-life factor concentrates, non-replacement therapies, and gene therapy, have substantially reduced bleeding frequency and improved life expectancy....
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Fibrin is principally involved in blood coagulation and hemostasis. Its structure, abundance and turnover are reciprocally related to blood flow. Decreased abundance of fibrin in afibrinogenemic or hemophilic patients leads to bone disorders such as bone cysts or hemarthroses. Fibrin accumulation in growing children with chronic disease leads to osteoarthritis and physeal disruption. Despite its clear role in bone disorders; the underlying mechanisms...
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