Priorities when deciding on participation in early-phase gene therapy trials for Duchenne muscular dystrophy: a best-worst scaling experiment in caregivers and adult patients

Paquin RS, Fischer R, Mansfield C, et al. Priorities when deciding on participation in early-phase gene therapy trials for Duchenne muscular dystrophy: a best-worst scaling experiment in caregivers and adult patients. Orphanet journal of rare diseases 2019;14:102   Abstract PURPOSE: Several gene therapy trials for Duchenne muscular dystrophy initiated in 2018. Trial decision making is […]