In a new transatlantic collaboration, Parent Project Muscular Dystrophy (PPMD) and Duchenne UK (DUK), the DMD Hub, and RTI International are conducting a study to explore attitudes towards gene therapy and gene editing from adults with Duchenne muscular dystrophy (Duchenne), parents and caregivers of children with Duchenne, and clinicians.

The 18-month study will also examine the tolerances, risks, and uncertainty for taking part in this new area of research.

An initial, first-of-its-kind, study was conducted by PPMD and RTI in 2017. The resulting data was instrumental in understanding perceived benefits and risk tolerance for emerging gene therapies, based on what was known at the time.

The community’s experience with and knowledge about gene therapy has since advanced through trials and research. Now, we plan to review the original survey and expand it to capture how people’s thoughts and feelings about this potential therapy has developed.

The study will be in two parts: the first will involve interviews with clinicians and focus groups with caregivers and adults with Duchenne. These discussions, along with input from the Stakeholder Advisory Board, will form the basis for developing the second stage survey. This survey will be shared with the patient community in both the US and UK.

Findings from the survey will inform discussions by regulators, pharmaceutical companies, and clinicians, ensuring that decisions about gene therapy are based on patients’ preferences. It will also highlight areas where PPMD, Duchenne UK, and the DMD Hub can provide support to the patient community moving forward.

Ryan Fischer, PPMD’s Chief Advocacy Officer, said:

“We are thrilled to be part of this unprecedented collaboration to explore patient and caregiver preferences in gene therapy. PPMD is pleased to see this research continue following our first study in 2017. Preferences must be measured over time as new information comes to light, to better understand what matters most to families as they make decisions about clinical trials and potential treatments.”

Alex Johnson and Emily Crossley, co-founders of DUK, said:

“It’s a privilege to pick up on the great work done by PPMD and RTI in 2017, and to continue to research patient preferences in gene therapy as the field evolves. The outcomes of this study will have global reach and be of use to all stakeholders in working out our response to these promising new medicines.”

The study began in late 2021 and we will invite the patient community in both the US and UK to participate in our survey later this year.