3 results
Primary Objective• To evaluate the efficacy of two dosage regimens of palovarotene compared with placebo in preventing new osteochondromas (OCs) in subjects with multiple osteochondromas (MO) due to exostosin 1 (Ext1) or exostosin 2 (Ext2) mutations…
Primary Objective* To compare the effect of a 30-week course of mavacamten with placebo on clinical response comprising of exercise capacity and clinical symptoms in participants with symptomatic obstructive hypertrophic cardiomyopathy (oHCM)…
Primary Objectives: • To assess the efficacy of a 48-week course of mavacamten compared to placebo on patient- reported health status (symptoms and physical limitations)• To assess the efficacy of a 48-week course of mavacamten compared to placebo…