A Placebo-controlled study of Maralixibat in Subjects with Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)
To find out the effects of Maralixibat on the treatment of PFIC.
To find out the effects of Maralixibat on the treatment of PFIC.
1) To determine if 5 doses of Epo (Erythropoietin) 1000 U/kg (birth weight) intravenous (IV) reduces the rate of death or neurodevelopmental impairment (mild, moderate, or severe) at 24 months of age.
2) To assess safety of Epo.
3) To determine whether Epo decreases the severity of HIE-induced brain injury as evidenced by early MRI and plasma biomarkers of brain injury.
This research is being done to test the safety and effectiveness of intranasal carbetocin to treat Prader Willi Syndrome.
To evaluate the efficacy of tofacitinib based on remission in pediatric participants with moderately to severely active Ulcerative Colitis (UC).