3 results
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…
The objective of this pivotal study is to assess the long-term safety, tolerability, pharmacokinetics and pharmacodynamics of RP103 in pediatric and adult patients with nephropathic cystinosis. Results of this Phase 3 study will be used to support…
The purpose of this study is to evaluate and document the safety information, how effective and cost effective the DTM SCS stimulation system is and the standard pain treatment in patients with chronic, difficult to treat back pain who are not…