4 results
The purpose of this study is to examine the efficacy and safety of 26 weeks treatment with inhaled mannitol in subjects with cystic fibrosis. Previous studies with inhaled mannitol have demonstrated improvements in lung function, mucociliary…
The primary objective is to compare18F-MFBG PET-CT imaging for neuroblastoma patients with the current standard of imaging, 123I-MIBG SPECT, using the SIOPEN score for skeletal lesions and the number of detected soft tissue lesions as endpoints.
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…
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…