Is more involvement needed in the clinical trial design & endpoints?

Excerpt

Duchenne muscular dystrophy (DMD) is a recessive X-linked form of muscular dystrophy, affecting around 1 in 3,600 boys, which results in muscle degeneration and eventual death. Long before any promising drug was at the horizon Duchenne parents came forward to organise research meetings where they made it clear they were willing to shoulder responsibility and contribute towards advancing treatments and a cure. They became funders of peer reviewed research and advocated for government support. Some organisations started their own research institutes others invested in extramural research, clinical centers and industry to develop viable treatments for DMD and BMD. Currently several potential drugs are in phase 3 trials. …