6 results
Main objective: To map the natural course of the visual and hearing deterioration in Usher Syndrome type 2 for upcoming genetic therapy studies.Secondary Objective 1): To determine a. the necessary type of (combined) examinations, b. the sample size…
1. To report the natural history of retinal degeneration in patients with biallelic mutations in the EYS gene.2. To identify sensitive structural and functional outcome measures to use for future multicenter clinical trials in EYS-related retinal…
The goal of this study is to characterize the natural course of IRDs that can potentially be modulated by future therapy. Second, this study aims to understand the relationship between various structural and functional biomarkers in potentially…
Primary Objectives: * to establish - which is the best treatment, MTX or baricitinib, to ensure rapid symptom relief of recent onset UA, based on clinical and patient reported outcomes from baseline to 3 months. Secondary Objective: * to establish…
The main objective of the proposed study is to investigate the effect of plasma products and albumin on markers of endothelial function in a human endotoxemia model.
To report the natural history of retinal degeneration in patients with biallelic mutations in the USH2A gene; to identify sensitive structural and functional outcome measures to use for future multicenter clinical trials in USH2A-related retinal…