Ionis Pharmaceuticals provided an update on its recently-concluded phase 1-2 clinical trial of IONIS-DMPK-2.5Rx, an experimental compound for the treatment of type 1 myotonic dystrophy (DM1). Analysis showed mixed results from the trial.
MDA is excited to announce that we are accepting applications for the inaugural National Community Advisory Committee, a group that will advise MDA on matters of importance to families living with neuromuscular diseases and help inform MDA’s efforts to support individuals to live unlimited.
When opportunity knocks, 15-year-old Justin answers. Born with CMD, Justin has traveled to China, been on a 500-mile bike ride across Iowa, and even modeled in a fashion show. How? Justin always says, ‘Yes.’ That’s how he lives unlimited.