Duchenne Muscular Dystrophy (DMD) is a rare disease caused by a genetic mutation. It effects approximately 1 in 3,500 -5,000 males born worldwide. It leads to the muscles in the body becoming weak and damaged over time and is eventually fatal.
The overall aim of this study was to evaluate the impact of a new IMP to treat the symptoms of DMD.
Recruitment of patients for the trial has been simpler than many others, as the site teams recognize the benefits of MRN’s Home Trial Support service to support this vulnerable patient population as they move through this suite of trials.
At site, multiple function, endurance, and capability tests are carried out, to evaluate muscle function status in the patients. In weekly home visits, MRN’s specialist healthcare professionals complete an IMP infusion and carry out regular vital signs and concurrent medication checks.