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.
Safety of Fecal Microbiota Transplantation (FMT) in children with Crohn's disease in remission.
To know if stool transplant from a healthy person into an ulcerative colitis patient can help treat the disease.
To determine the safety and feasibility of autologous umbilical cord blood-derived mononuclear cells delivered into the myocardium of the right ventricle during planned Stage II Glen surgical procedure for individuals with hypoplastic left heart syndrome.
To evaluate the efficacy of tofacitinib based on remission in pediatric participants with moderately to severely active Ulcerative Colitis (UC).
To assess the survival rate of subjects at 6 and 12 months following implantation of the Bioabsorbable pulmonary valve conduit. To assess the rate of conduit failures at 6 months, requiring re-intervention or reoperation.