PPS - RTI
Duchenne UK
RTI International
This is a collaboration funded by Duchenne UK. It involves RTI International, Parent Project Muscular Dystrphy and Newcastle University, who are all recepients of funding based on their contribution. This collaborative project will assess and explore clinician, patient, and caregiver attitudes, priorities, and risk tolerance regarding gene transfer technologies. These technologies offer great promise to be disease modifying but come with risks and burden. The study will employ a research and community engagement approach previously developed by Holly Peay at RTI and the collaborating Parent Project Muscular Dystrophy (PPMD) team. Instruments from the prior PPMD gene therapy preference study will be revised to meet current state of the science and will be reused in the proposed study. Data will be collected in the United States and the United Kingdom. Data on attitudes, priorities, and risk tolerance will be compared across the countries.
7.1 Individual care needs