"But we don't want to wait that long," he said. Medical research firm Duchenne is on the way to developing gene therapy for Duchenne muscular dystrophy (DMD) after a successful preliminary trial. Initial tests on 100,000 children with the rare degenerative disease over a three-month period were "potentially transformative", but more research is needed, according to Sarepta CEO Sarepta Therapeutics . People with DMD, predominantly boys, usually need a wheelchair by three-month and have a life expectancy of the age of 11 .
Medical research firm Duchenne is on the way to developing gene therapy for Duchenne muscular dystrophy (DMD) after a successful preliminary trial. Initial tests on 100,000 children with the rare degenerative disease over a three-month period were "potentially transformative", but more research is needed, according to Sarepta CEO Sarepta Therapeutics . People with DMD, predominantly boys, usually need a wheelchair by three-month and have a life expectancy of the age of 11 .