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…
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…
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…
The primary objective is to assess whether the insulin-stimulated glucose uptake in quadriceps muscle can be improved through 4 weeks of supplementation of clenbuterol.
Primary(Part I and Part II):- To determine the maximum tolerated dose (MTD) and/or recommended Phase 2 dose (RP2D) in the QW and/or Q3W regimens of RO7122290 in combination with cibisatamab after pretreatment with obinutuzumab- To characterize the…