News

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,…

Ameritas Honors Hemophila A Advocate With $20K Grant to NHF

Jim Christensen Jr., a hemophilia A patient who has supported the National Hemophilia Foundation (NHF) for more than three decades, has received the Lester A. Rosen Humanitarian and Achievement Award, given annually by Ameritas, for which he is an independent financial representative. With the award, the insurance company…

Potential Gene Therapy GENV-HEM Earns Orphan Drug Status

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to GENV-HEM, GeneVentiv Therapeutics’ investigational gene therapy for hemophilia A and B patients, with or without clotting factor inhibitors. Orphan drug designation is given to treatment candidates that have the potential to be safe and effective…

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,…

8-Week Exercise Program Yields Multiple Benefits

Hemophilia patients experienced improvements in joint health, mobility, and overall quality of life after completing a supervised eight-week exercise program, a study reports. However, in order to achieve long-lasting benefits, regular exercise may be required. The study, “Effects of a supervised therapeutic exercise program on musculoskeletal health and…