Sarepta Closer To Muscular Dystrophy Wonder Drug

(RTTNews) - Shares of Sarepta Therapeutics Inc., (SRPT) is currently trading up over 33% after the company released positive preliminary data for its experimental gene therapy for patients with Duchenne muscular dystrophy.

The company released positive preliminary results from its Phase 1/2a gene therapy clinical trial assessing micro-Dystrophin in three children with Duchenne muscular dystrophy.

All patients showed robust expression of transduced micro-dystrophin, which is properly localized to the muscle sarcolemma, as measured by immunohistochemistry. Mean gene expression, as measured by percentage of micro-dystrophin positive fibers was 76.2% and the mean intensity of the fibers was 74.5% compared to normal control.

All patients showed significant decreases of serum creatine kinase levels, with a reduction of over 87%. Creatine kinase is an enzyme associated with muscle damage and patients with DMD exhibit high levels of it.

"I have been waiting my entire 49-year career to find a therapy that dramatically reduces CK levels and creates significant levels of dystrophin," Dr. Jerry Mendell of Nationwide Children's Hospital said in a statement. "Although the data are early and preliminary, these results, if they persist and are confirmed in additional patients, will represent an unprecedented advancement in the treatment of DMD."

Solid Biosciences is also developing a gene therapy for DMD. The company on Monday reported that the Food and Drug Administration had released the hold on its clinical trial program.

SRPT is currently trading at $140.40, up $35.16 or 33.41%, on the Nasdaq.