The goal of this study is to collect additional information on the safety of long-term treatment with AGAMREE® and to explore long-term clinical impact of AGAMREE® on quality of life, as assessed by standardized patient-reported outcome measures (QoL questionnaires) in male patients aged 2 years and older with Duchenne muscular dystrophy (DMD).
Currently recruiting: the trial is enrolling patients. A data readout is not expected until after enrollment closes and the follow-up period is complete.
Catalyst Pharmaceuticals (CPRX) is the sponsoring company for this trial. BiotechSign currently grades this company F (34/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.