Re-evaluating Acceptable Risk of Death from Gene Therapy: A Threshold Study Among Individuals with Duchenne Muscular Dystrophy and Their Caregivers in the US and UK
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Patient and caregiver treatment preferences should be incorporated into the drug development process. We updated a 2018 survey to reflect current knowledge about gene therapy for Duchenne muscular dystrophy (DMD) and obtained new data in the U.S., U.K., and ot... ...
