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.
To collect information on the long-term effects of XLH on children and adults who have participated in burosumab clinical trials, adults and children who have not participated in burosumab clinical trials, and who may or may not have been treated with burosumab during the course of the study.
This study will evaluate the effect of Denosumab on lumbar spine mineral density (BMD), as assessed by dual-energy X-Ray absorptiometry (DEXA) at 12 months in children 5 to 17 years of age with GIOP.
This research is being done to test the safety and effectiveness of intranasal carbetocin to treat Prader Willi Syndrome.