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.
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Denosumab treatment for glucocorticoid-induced osteoporosis.
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.
Prader-Willi Syndrome (PWS)
This research is being done to test the safety and effectiveness of intranasal carbetocin to treat Prader Willi Syndrome.