Diagnosis & Disease Information

Researchers sought to determine whether gene therapy with valoctocogene roxaparvovec would improve health-related QoL for men with severe hemophilia A.

Researchers sought to determine whether prophylactic EHL factor VIII or IX concentrates would improve adherence rates and outcomes in patients with hemophilia.

Researchers sought to determine treatment adherence in people with severe hemophilia.

Researchers sought to determine characteristics that predict the use of diagnostic PGT or PND for hemophilia carriers.

Researchers sought to determine whether simoctocog alfa would be safe and effective for bleeding prophylaxis patients with severe hemophilia A.

Researchers sought to determine whether rurioctocog alfa pegol prophylaxis would be effective for reducing ABRs in patients with hemophilia A.

Researchers sought to determine factors that prevent patients with hemophilia from engaging in physical activity.

Researchers sought to determine the coagulation potential of emicizumab in plasmas obtained from infants and toddlers with severe hemophilia A.

Researchers sought to determine the cost-effectiveness of prophylactic emicizumab in patients with moderate or mild hemophilia A without inhibitors in the United States.