Showing 668 results for "replacement therapy"

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Catalyst Halts MarzAA Program for Hemophilia, Despite Positive Results

In an unexpected turn, Catalyst Biosciences is discontinuing the clinical development of marzeptacog alfa activated (MarzAA), its experimental under-the-skin therapy for hemophilia A and B patients with inhibitors, which was being evaluated in an international Phase 3 clinical trial. “Based on several factors including a recently updated feasibility assessment,…

Patients Express Gains in Switch to Extended Half-life Therapies

For some patients with moderate or severe hemophilia, switching from standard to extended half-life replacement therapy results in improvements in key outcome measures, a study in Canada found. Most of these self-reported gains in health-related quality of life, and physical, mental, and social functioning three months after the switch,…

Implanting 3D Bioscaffolds in Rats Works to Trigger FVIII Production

Implanting bioscaffolds — 3D biological scaffolds made up of collagen and other molecules — carrying transformed liver cells into rats successfully triggered the production of factor VIII (FVIII), the clotting protein missing in people with hemophilia A. According to researchers, these bioscaffolds may be a therapeutic alternative to FVIII…

‘Reverse Vaccination’ May Help Prevent Immune Response

A newly designed platform based on the principle of “reverse vaccination” could be used to prevent the development of an immune response that blocks the effectiveness of treatments for hemophilia and other disorders. “For nearly a third of patients with hemophilia A … their own immune system is…

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…