Investment in gene therapy has continued to pay off as Sarepta Therapeutics recently announced positive results from phase 1/2a trial with its experimental treatment for patients with Duchenne Muscular Dystrophy.
Duchenne muscular dystrophy is a rare and fatal genetic disease that causes muscle degeneration and is identified by elevated levels of creatine kinase in the bloodstream. Patients who received Sarepta’s drug experienced a significant reduction of their creatine kinase levels.
Although the results are preliminary, it represents real progress towards a long awaited and viable treatment option.
I have been waiting my entire 49-year career to find a therapy that dramatically reduces [creatine kinase] levels and creates significant levels of dystrophin