7 results
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: - Clinical: To assess the efficacy of MPDL3280A pre-cystectomy with respect to pCRR in patients with T2-T4aN0M0 transitional cell carcinoma of the bladder- Biological: To assess the effect of 2 x 3 weekly cycles of MPDL3280A pre-…
The overall aim is develop a rational dosing regimen of liposomal amphotericin B in obese patients. To achieve this goal we have defined the following objectives: Primary objective:* To determine the pharmacokinetics of liposomal amphotericin B…
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…
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…
To assess the feasibility of a larger study comparing HFNO with NIV as first line treatment in hypercapnic, acidotic AECOPD.