4 results
Primary:To assess the effect of bilateral treatment with AAV5-hRKp.RPGR on functional vision as measured by vision-guided mobility assessment.Secondary:To assess changes after treatment administration in retinal function, functional vision, visual…
Primary Objective:• To evaluate the effects of withdrawal of tiratricol treatment (placebo group) on serum total triiodothyronine (T3) concentrations, measured by liquid chromatography with tandem mass spectrometry (LC/MS/MS), and the requirement…
Main objective:To assess the long-term safety and tolerability of AAV5-hRKp.RPGR in individuals with RPGR-XLRPTo assess the long-term efficacy of treatment with AAV5-hRKp.RPGR in individuals with RPGR-XLRP based on assessments of functional vision…
Part I:Primary Objective: Evaluate the effects of tiratricol on neurodevelopment in young MCT8 deficiency patients, as measured by the Gross Motor Function Measure (GMFM)-88 and Bayley Scales of Infant Development (BSID-III) Gross Motor Skill Domain…