Systemic diseases of liver origin (SDLO) are complex diseases in multiple organ systems,
such as cardiovascular, musculoskeletal, endocrine, renal, respiratory, and sensory organ …

Non-compressible hemorrhage is an unmet clinical challenge, which occurs in inaccessible
sites in the body where compression cannot be applied to stop bleeding. Current treatments …

Background Prophylactic factor replacement in patients with hemophilia B improves
outcomes but requires frequent injections. A recombinant factor IX Fc fusion protein (rFIXFc) …

This multinational, randomized, single-blind trial investigated the safety and efficacy of
nonacog beta pegol, a recombinant glycoPEGylated factor IX (FIX) with extended half-life, in …

Replacement therapy with factor IX (FIX) concentrates is the recommended treatment for
patients with hemophilia B, an X-linked bleeding disorder occurring in 1: 25 000 male births …

The pharmacokinetic (PK) response to factor VIII (FVIII) and factor IX varies between patients
and this has important clinical implications for treatment. Although PK is affected by patient …

Current management of hemophilia B entails multiple weekly infusions of factor IX (FIX) to
prevent bleeding episodes. In an attempt to make a longer acting recombinant FIX (rFIX), we …

A surge in therapeutic clinical trials over recent years is paving the way for transformative
treatment options for patients with hemophilia. The introduction of recombinant factor …

Thrombotic adverse events (AEs) after clotting factor concentrate administration are rare but
the actual rate is unknown. A systematic review of prospective studies (1990–2011) …

Abstract Adeno-associated virus (AAV)-mediated gene therapy is a novel treatment
promising to reduce morbidity associated with hemophilia. Although multiple clinical trials …