The goal of this international observational study is to learn about the natural history of Danon disease in male patients (≥8 years of age) and female patients (8 to 50 years of age).
Currently recruiting: the trial is enrolling patients. A data readout is not expected until after enrollment closes and the follow-up period is complete.
Rocket Pharmaceuticals (RCKT) is the sponsoring company for this trial. BiotechSign currently grades this company C (60/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.