Abstract
In 2017/2018, we concluded a study on risk tolerance for gene therapy (GT) for Duchenne muscular dystrophy (Duchenne). Duchenne is a progressive, fatal condition. GTs under trial could be disease modifying, not curative. GT has serious risks, including risk of death. In our prior study we assessed maximum acceptable risk (MAR) of death in U.S.-based adults with Duchenne and parents. In GT trials over the past 5 years there were serious adverse events, clinical holds, and one death in late 2021. We initiated a second study to assess current MAR in the U.S., United Kingdom, and to explore experiences and attitudes of clinicians.
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