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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 study is a randomized, double-blind, active-controlled, titrated, parallel arm, multicenter study. It will compare the efficacy, safety and tolerability of twice daily Chronocort with twice daily IRHC (Cortef®) over a randomized treatment period of up to 52 weeks in participants aged 16 years and over with known classic CAH due to 21-hydroxylase deficiency. The primary efficacy assessment of biochemical responder rate and the key secondary assessments of dose responder rate and mean total daily dose will be assessed after 52 weeks of randomized treatment.

To find out safety and effectiveness of dupilumab (Dupixent) in infants with severe eczema.

To find out the effects of dupilumab (Dupixent) on skin infection in eczema.

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.

Hydroxychloroquine is given as an investigational drug to relatives of someone with type 1 diabetes who have positive diabetes antibodies in an attempt to prevent or delay the onset of type 1 diabetes. See website for more details https://www.trialnet.org/our-research/prevention-studies/hydroxychloroquine-hcq

This is a Phase 3, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered twice daily (bid) with breakfast and evening meals for28 weeks in approximately 81 pediatric subjects with classic congenital adrenal hyperplasia (CAH) due to21-hydroxylase deficiency. Eligible subjects will be randomly assigned in a 2:1 ratio (active:placebo) to either crinecerfont (25 mg bid via oral solution for subjects 10 to <20 kg, 50 mg bid via oral solution for subjects 20 to <55 kg, or 100 mg bid via oral capsules for subjects ≥55 kg) or matching placebo (oral solution placebo for subjects <55 kg and oral capsule placebo for subjects ≥55 kg). Dose assignment from Day 1 to Week 28 will be based on the subject’s weight at Day 1. After the 28-week placebo-controlled treatment period, there will be a 24-week, open-label treatment period, during which all subjects will receive crinecerfont at doses based on their Week 28 body weight.

This research is being done to test the safety and effectiveness of intranasal carbetocin to treat Prader Willi Syndrome.