7 results
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 evaluate RBC transfusion independence (RBC-TI) of luspatercept compared with placebo for the treatment of anemia due to IPSS-R very low, low, or intermediate risk MDS in subjects with ring sideroblasts (* 15%) who…
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…
Primary objective:• To evaluate the efficacy of luspatercept on RBC transfusion independence (RBC-TI for 12 weeks [84 days] with an associated concurrent mean hemoglobin increase >= 1.5 g/dL) compared with epoetin alfa for the treatment of…
PART A and PART DTo assess the safety and tolerability of TAK-994 following multiple oral doses in subjects with narcolepsy with or without cataplexy (NT1 or NT2).PART B and PART C To assess the efficacy of TAK-994 on reducing excessive daytime…