Patients’ Priorities for Gene Therapy: Durability and Bleeding Control

Patients’ Priorities for Gene Therapy: Durability and Bleeding Control

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Patients’ Priorities for Gene Therapy: Durability and Bleeding Control

Effectiveness in controlling bleeds and the potential for less frequent use of a treatment are two priorities people with hemophilia place on a potential gene therapy, and both ranked slightly higher than safety, a survey study of patients in the U.S. reports. The study, “Patient preferences and priorities for haemophilia gene therapy in the US: A discrete choice experiment,” was published in the journal Haemophilia. Hemophilia is caused by mutations in genes that encode proteins needed…

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