Gene Therapy AMT-060 for Hemophilia B Gains PRIME Designation in Europe
UniQure‘s investigational gene therapy AMT-060 for severe hemophilia B has received PRIME designation by the European Medicines Agency (EMA).
Articles by Margarida Azevedo, MSc
Hemophilia Federation Symposium Presents ‘Hope Ignites: Stories to Inspire’
The lives of four inspiring patients with bleeding disorders were introduced at the “Hope Ignites: Stories to Inspire” patient program…
South Korea Approves Catalyst’s IND Application for Hemophilia B Blood-clotting Treatment
South Korea has approved an Investigational New Drug (IND) application for Catalyst Biosciences’ experimental therapy CB 2679d/ISU304, a highly…
Hemophilia B Potential Treatment Refixia Receives Positive Opinion from the European Regulatory Agency
Novo Nordisk’s Refixia (nonacog beta pegol, N9-GP) received a positive opinion by the Committee for Medicinal Products for Human…
Hemophilia B Treatment SPK-9001 Wins Access to EMA’s PRIority MEdicines Program
The European Medicines Agency (EMA) will include SPK-9001 — an investigational drug for patients living with hemophilia B —  in Priority Medicines…
World’s Largest Genetic Hemophilia Research Repository Opens to U.S. Researchers
My Life, Our Future (MLOF), a national program founded by leaders in the hemophilia and blood disorder community, marked Feb.
Patient in Emicizumab Clinical Trial Dies, but It Appears Not to Be from Treatment
A hemophilia patient has died in a Phase 3 trial evaluating Roche’s  experimental treatment emicizumab (ACE910), raising further concern about its…
Orphan Drug Act May Be Helping Pharmaceuticals More Than Patients, Analysis Argues
An analysis was highly critical of the Orphan Drug Act, used by the U.S. Food and Drug Administration since 1983 to stimulate…
Plant-Based Hemophilia Therapy Tested in Dogs Shows Promise, Says Study
Researchers have developed a protein drug that may teach the body to tolerate, rather than reject, the clotting factor treatment given…
Lacking Employment, College Education Negatively Impacts Hemophilia Patients, Study Finds
Being unemployed or under-educated can have a negative effect on hemophilia patients’ quality of life, including their pain levels and…