4 results
To demonstrate the efficacy of repeated daily doses of 40 µg/kg/day and 120 µg/kg/day A4250 in children with progressive familial intrahepatic cholestasis (PFIC) Types 1 and 2, as determined by the following:* Proportion of patients experiencing at…
NAM, well known as a dietary supplement, has also been extensively studied in humans in a variety of diseases in both children and adults. However, the bioavailability of NAM in patients with JIA at the side of inflammation, and therefore it*s…
NAM, well known as a dietary supplement, has also been extensively studied in humans in a variety of diseases in both children and adults. However, the safety, tolerability of NAM in children with JIA is yet unknown. Furthermore, studies performed…
The primary objective is to compare the objective response rate (ORR) per Response Assessment in Neuro Oncology (RANO) criteria assessed by independent review committee (IRC) of DAY101 monotherapy versus standard of care (SoC) chemotherapy in…