Author Archives: Marta Figueiredo PhD

Research Explores Umbilical Stem Cells as Treatment

Using a baby’s own umbilical cells as vehicles to deliver factor VIII (FVIII) — the missing or defective clotting protein in hemophilia A — may be an effective, long-lasting, and more affordable therapeutic approach for this blood disorder, an early study suggests. The findings “lay the groundwork for future…

New Guidelines Issued to Self-administer Hemlibra

The National Hemophilia Foundation (NHF), through its Nursing Working Group (NWG), has released new guidelines to help hemophilia A patients self-administer Hemlibra (emicizumab). In its announcement, the foundation also highlighted the issuance of new guidelines for nurses to ensure safe and effective administration of standard into-the-vein…

Gene Therapy SPK-8011 Prevents Bleeds Up to 4 Years

A single dose of Spark Therapeutics’ investigational gene therapy SPK-8011 safely and effectively prevented bleeding episodes and the use of clotting factor VIII (FVIII) for up to four years in men with hemophilia A, according to the latest data from a Phase 1/2 trial and its extension study.

BioMarin Resubmits Roctavian Gene Therapy for EU Approval

BioMarin Pharmaceutical has resubmitted a regulatory application seeking approval in Europe for its experimental gene therapy Roctavian (valoctocogene roxaparvovec) for severe hemophilia A. That resubmission, to the European Medicines Agency (EMA), included one-year follow-up data from BioMarin on the therapy’s safety and effectiveness in treating the rare genetic…