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The results “meaningfully beat even the highest of expectations,” J.P. Morgan analyst Anupam Rama wrote in a note to clients.
Patients showed an average reduction of more than 87 percent in creatine kinase levels on day 60 – creatine kinase is an enzyme found within muscle cells that flow into the bloodstream due to muscle damage. Elevated creatine kinase is an indicator for Duchenne muscular dystrophy.
“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.”
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