FDA Grants Orphan Drug Status for ACE-083 to Treat Muscular Dystrophy
ACE-083 is designed to improve FSHD patients' strength and muscular growth
One of the main characteristics of FSHD is the progressive weakening of muscles. ACE-083 is designed to pinpoint and concentrate on targeted areas to improve strength and promote muscular growth. This drug is supposed to be injected into the areas where the patient is suffering the most.
During the initial phases of the trial, patients had an MRI before and after the drug was administered. The results showed that the muscles grew stronger and that the patients were able to move that area. Other than a few minor reactions around the injection sites, there were no side effects reported from the trials.
