3 results
The primary objective of this study is:* To assess the safety and tolerability of TPN-101 in patients with C9ORF72 ALS/FTDThe exploratory objectives of this study are:* To assess the concentrations of TPN-101 in plasma and cerebrospinal fluid (CSF…
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…