3 results
The primary objective of this study is to assess the efficacy of Ecopipam to reduce self-injurious behaviors (SIB) in adults and children with Lesch-Nyhan Disease (LND) in an outpatient setting. The secondary objectives of this study are to assess…
Our objective is to find predictive biomarkers for disease progression in RVCL-S, a monogenetic small vessel disease, and to dissect disease mechanism to identify new treatment targets to improve health for RVCL-S patients, which may also serve as…
Primary Objectives:• To assess the pharmacokinetics (PK) of bempedoic acid (ETC-1002 and ESP15228) in pediatric patients (6 to 17 years of age) with heterozygous familial hypercholesterolemia (HeFH) treated for 8 weeks.Secondary Objectives:• To…