He said if results of larger tests are also positive, then the company can seek FDA approval. Medical research firm Sarepta Therapeutics is on the way to developing gene therapy for Duchenne muscular dystrophy (DMD) after a successful preliminary trial. Initial tests on three children with the rare degenerative disease over a three-month period were "potentially transformative", but more research is needed, according to Sarepta CEO Douglas Ingram. The company has already received approval from regulators in Europe and plans to begin human trials later this year.
Medical research firm Sarepta Therapeutics is on the way to developing gene therapy for Duchenne muscular dystrophy (DMD) after a successful preliminary trial. Initial tests on three children with the rare degenerative disease over a three-month period were "potentially transformative", but more research is needed, according to Sarepta CEO Douglas Ingram. The company has already received approval from regulators in Europe and plans to begin human trials later this year.