Factor IX Grifols® is an effective and safe Factor IX concentrate and can be considered as a first line option for replacement therapy in haemophilia B patients. “
“This chapter contains sections titled: Introduction Lipid-enveloped viruses Nonlipid-enveloped viruses Prions Outlook References “
“Summary.  Eighteen cryoprecipitate

minipools, each made of 30 units of low volume, concentrated cryoprecipitate, have been treated by solvent-detergent and filtration (S/D-F) in a single-use CE-marked bag system. The S/D-F cryoprecipitate contained a mean of 10.5 IU mL−1 factor VIII (FVIII), 17 mg mL−1 clottable fibrinogen, and >10 IU mL−1 von Willebrand factor ristocetin BVD-523 cost co-factor, and anti-A and anti-B isoagglutinins were undetectable. The products have been infused in 11 severe (FVIII <1%) haemophilia A patients (mean age: 17.4 years; mean weight: 57.6 kg) at a dose close to 40 IU kg−1. Patients were hospitalized for at least 36 h to determine FVIII recovery, half-life and Barasertib mouse clearance. They were also closely monitored for possible adverse events. None of the infused patients demonstrated reactions or adverse events even though they did not receive anti-allergic drugs or corticosteroids prior to infusion. The mean recovery of FVIII 10 min postinfusion

was 69.7%. Mean FVIII half-life was 14.2 h and clearance was 2.6 mL h−1 kg−1. All patients had a bleeding-free interval of 8–10 days postS/D-F cryoprecipitate infusion. The data show that S/D-F cryoprecipitate Lonafarnib mouse FVIII presents a normal pharmacokinetics profile, and support that it could be safely used for the control of acute and chronic bleeding episodes

in haemophilia A patients. “
“The use of induced pluripotent stem cells (iPSCs) as an autologous cell source has shed new light on cell replacement therapy with respect to the treatment of numerous hereditary disorders. We focused on the use of iPSCs for cell-based therapy of haemophilia. We generated iPSCs from mesenchymal stem cells that had been isolated from C57BL/6 mice. The mouse iPSCs were generated through the induction of four transcription factor genes Oct3/4, Klf-4, Sox-2 and c-Myc. The derived iPSCs released functional coagulation factor VIII (FVIII) following transduction with a simian immunodeficiency virus vector. The subcutaneous transplantation of iPSCs expressing FVIII into nude mice resulted in teratoma formation, and significantly increased plasma levels of FVIII. The plasma concentration of FVIII was at levels appropriate for human therapy at 2–4 weeks post transplantation. Our data suggest that iPSCs could be an attractive and prospective autologous cell source for the production of coagulation factor, and that engineered iPSCs expressing coagulation factor might provide a cell-based therapeutic strategy appropriate for haemophilia. “
“Summary.  Hemophilia A is an X-linked, inherited, bleeding disorder caused by the partial or total inactivity of the coagulation factor VIII (FVIII).