3 results
the objective of the current study is to evaluate the safety and efficacy of enzyme therapy in the Dutch population of patients with MPS I, II and VI; to compare this to the natural course of these diseases; to make an inventory of the direct and…
Primary objective of the study:* To compare overall survival (OS) in the General Population patients treated with BBI-608 plus biweekly FOLFIRI (Arm 1) versus biweekly FOLFIRI (Arm 2)* To compare OS in the pSTAT3-positive (pSTAT3(+)) Subpopulation…
In patients with symptomatic New York Heart Association (NYHA) Class II/III/IV dilated cardiomyopathy (DCM) in which mutations in the gene encoding the lamin A/C protein (LMNA) have been implicated:Primary Objective:* NYHA Class II/III patients only…