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…
Main study: to examine whether low dose GC therapy is more effective via the oral route compared to the parenteral route.Dose finding study (pilot study): to examine which oral GC dose is needed for a noticeable decrease in Erythrocyte Sedimentation…
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…