7 results
The aim of this study is to improve 6MP treatment in pediatric leukemia patients, by developing and licensing a pediatric liquid formulation of 6MP, assessing its stability and bioequivalence, and ensuring a nationwide introduction of the new…
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…
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 PK of MPhs and MP.
The primary objective of this clinical trial is to obtain pilot safety and efficacy data on treatment of PAH patients by 6-MP. The secondary objective of this clfnical trial is to determine whether LEPs transcriptome analysis will identify a subset…
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…