Tag: Gene Therapy
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
Breyanzi Approved for Certain Types of Large B-Cell Lymphoma
The CAR T cell therapy OK'd for patients who have not responded to or have relapsed after receiving two or more other systemic treatments
Medicare to Cover CAR-T Therapy for Leukemia, Lymphoma
Chimeric antigen receptor T-cell therapy boosts a patient's own immune cells
FDA: Approval of Zolgensma Was Based on Manipulated Data
Despite inaccurate data, FDA says it is 'confident that Zolgensma should remain on the market'
Lifesaving Drug for Infants Costs $2.1 Million a Dose
When Zolgensma was approved, maker Novartis said it expected insurance companies to cover the cost
