6 results
-Evaluate the long-term safety and tolerability of apitegromab in patients with Type 2 and Type 3 SMA-Evaluate the long-term efficacy of apitegromab by assessing changes in motor function outcome measures at prespecified time points-Further evaluate…
Primary objective: to investigate the effect of amiloride on urine volume and urinary concentrating ability in patients on chronic lithium therapy suffering from polyuria.We hypothesize that amiloride therapy will improve urinary concentating…
The primary objectives1. To assess the safety and tolerability of intravenous tracer administration of ANXV-800CW in up to three doses (0.5 mg, 1.0 mg, 2.0 mg flat dose) in patients with RVO and/or DR2. To determine the feasibility of molecular…
Primary-Assess the efficacy of apitegromab compared with placebo using the HFMSE in patients 2 through 12 years oldKey secondary-Assess the efficacy of apitegromab compared with placebo based on the number of patients with clinical improvement in…
The aim of this pilot study is to further unravel the pathophysiologic mechanism of NS-induced hypercholesterolemia which willfurther guide the treatment of patients with NS.Given the recent insights on PCSK9-ENaC inhibition, our hypothesis is that…
We aim to investigate if arterial stiffness is exacerbated due to a high-salt diet in patients with ADPKD. We also intend to explore whether treatment with amiloride prevents the effect of high salt on arterial stiffness.