Description
Seeking boys between the ages of four and 17 with Duchenne Muscular Dystrophy (DMD) for a study of an investigational gene therapy drug that may improve their body's ability to make dystrophin, a protein that helps protect muscles from stress and damage.
Overview
We are studying how safe and whether the investigational therapy has the potential to treat certain boys with DMD. The study has two parts, a screening period and a dosing and follow-up period. Each volunteer who completes the screening will receive the investigational drug at either a high or low dose. Boys cannot be on certain medications or supplements while on the study.
What we're hoping for
We hope that the investigational drug will be safe to use and slow muscle damage and reduce the overall loss of muscle function in boys with DMD
Additional Information
ClinicalTrials.gov Identifier: NCT03368742