3 results
Not approvedWill not start
To induce a vaccine specific immune response, hereby we hope to introduce the possibility of a new treatment to improve patient outcome and survival.
Approved WMOCompleted
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)…
Approved WMORecruiting
To evaluate safety, tolerability pharmacokinetic profile; to explore pharmacodynamics; to evaluate major effects of Guanabenz on quantitative MRI and clinical parameters in pediatric patients with VWM; explore biomarkers in blood and cerebrospinal…