Tag: Gene Therapy
FDA Advisers to Weigh New Gene Therapies for Sickle Cell Anemia
Advisers will consider whether more research is needed into potential unintended consequences of the new therapies
Racial, Ethnic Differences ID’d for Idecabtagene Vicleucel in Multiple Myeloma
Non-Hispanic Black patients more likely to develop cytokine release syndrome; best overall response rate was lower in Hispanics
FDA Approves First Gene Therapy for Severe Hemophilia A
Roctavian is a viral vector that carries the gene for factor VIII
FDA Approves First Gene Therapy to Treat Duchenne Muscular Dystrophy
The recombinant gene therapy is administered in a single intravenous dose
Gene Therapy Not Cost-Effective for Sickle Cell by Conventional Measures
The inequality aversion parameter would need to be 0.90 for gene therapy to be preferred per distributional cost-effectiveness analysis standards
Benefits of Valoctocogene Roxaparvovec Persist in Hemophilia A
The mean annualized treated bleeding rate decreased by 84.5 percent from baseline to 104 weeks
Gene Therapy Beneficial for Patients With Hemophilia B
One infusion of etranacogene dezaparvovec noninferior and superior to factor IX prophylaxis
FDA Approves First Cell-Based Gene Therapy for Beta-Thalassemia
The single-dose treatment is approved for transfusion-dependent adults and children
Gene Therapy Promising for SCID Due to ADA Deficiency
High overall, event-free survival seen at 24 and 36 months for severe combined immunodeficiency due to ADA deficiency
Trial of Gene Therapy for Sickle Cell Disease Halted
Two patients participating in the study developed cancer years after treatment
