The impact of capping health system cost savings on the projected cost-effectiveness of etranacogene dezaparvovec compared with factor IX prophylaxis for the treatment of hemophilia B. [PDF]
Sarker J+4 more
europepmc +1 more source
Human umbilical cord mesenchymal stem cell-based gene therapy for hemophilia B using scAAV-DJ/8-LP1-hFIXco transduction. [PDF]
Bu Z, Lou J, Xu W, Zhang L, Tang Y.
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Images of Temporomandibular Joint Arthropathy in Patient with Severe Hemophilia B with Inhibitor and Patient with Type III von Willebrand Disease with Inhibitor [PDF]
Yenel S+4 more
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Nonacog beta pegol prophylaxis in children with hemophilia B: safety, efficacy, and neurodevelopmental outcomes for up to 8 years. [PDF]
Walsh KS+6 more
europepmc +1 more source
A Post-Authorization Safety Surveillance Study to Report Clinical Experience with Purified Factor IX Concentrate in Pediatric Patients with Hemophilia B. [PDF]
Igrutinović Z+6 more
europepmc +1 more source
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AbstractHemophilia B (HB) is an X-linked bleeding disorder caused by deficiency of factor IX (FIX). Patients with the severe form (FIX <1%) account approximately for 30 to 45% of persons with HB and usually suffer from recurrent joint, soft-tissue, and muscle bleeds.
Erminia Baldacci+6 more
openaire +4 more sources