3 results
The primary objective is to compare the efficacy of palifermin relative to placebo when given either pre-and post-high dose chemotherapy or pre-high dose chemotherapy only with regard to the severity of oral mucositis.
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…