Author Archives: Marta Figueiredo PhD

Optimal Disease Control Advised for Hemophilia B Patients

People with severe hemophilia B are likely to develop hemophilic arthropathy — a painful and degenerative joint disease caused by recurrent bleedings — despite the lower bleeding frequency relative to hemophilia A, a natural history study shows. The data also highlights that prophylactic (preventive) treatment guidelines are not being…

uniQure’s Hemophilia B Gene Therapy Program Placed on Hold

The U.S. Food and Drug Administration (FDA) has placed a clinical hold on uniQure’s gene therapy program for hemophilia B, which includes AMT-061 (etranacogene dezaparvovec), due to a possibly related serious adverse event in one patient. The event concerns a preliminary diagnosis of hepatocellular carcinoma (HCC), a form…