3 results
Primary objective:To evaluate the efficacy of soraprazan in reducing the amount of lipofuscin inRPE cells of subjects with Stargardt disease by assessing the change inquantitative auto-fluorescence (qAF8) from baseline to after treatment…
* To decrease mortality in MS-LCH by an early switch of patients with risk organ involvement, who do not respond to front-line therapy, to a more intensive treatment (Stratum III or Stratum IV).* To reduce reactivation rates and permanent…
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…