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.