Neurology® published the results of the HOPE-Duchenne clinical trial that evaluated the safety and efficacy of a single intracoronary dose of Capricor Therapeutics. The Phase I/II, randomized, controlled, open-label trial found that CAP-1002 demonstrated improvement in cardiac muscle function and reduction in cardiac scarring that were statistically-significant and sustained improvement of skeletal muscle functions in patients with Duchenne muscular dystrophy, a fatal genetic disease with limited treatment options. The HOPE-Duchenne trial also found no serious safety issues. The HOPE-Duchenne clinical trial enrolled 25 patients and was conducted at three centers in the U.S. All participants had significant cardiac scarring and approximately two-thirds were wheelchair-dependent at the time they began the trial. During the 12-month course of the trial, all patients received the standard-of-care for Duchenne muscular dystrophy, including oral steroids, and 13 also received one dose of intracoronary CAP-1002 (75 million cells) upon randomization. Using the Performance of the Upper Limb (PUL) test, which is a validated functional assessment of upper limb activities of daily living (ADL), the researchers found sustained improvement in eight of the nine CAP-1002 treated patients in the mid-distal PUL, which was manifested by improvements in the patients’ abilities to use their arms and hands. The control patients had no improvement in the mid-distal PUL tests.