Over a 6-month period, adolescents and their parents will participate in clinical interviews, EEG tasks, and surveys about depression, mental health, and friendships, among other topics. Over time, we hope to understand depression and mental health outcomes in autistic adolescents with intellectual disability (14-17 years old) and their parents.
To explore the safety and treatment effect of intravenous (IV) Remodulin as add on therapy in neonates with PPHN compared to placebo.
This is a Phase 3, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, and tolerability of NBI-74788 versus placebo administered bid with breakfast and the evening meal (doses separated by approximately 12 hours) for 24 weeks in approximately 165 adult subjects with classic CAH due to 21-hydroxylase deficiency.
This is a randomized, double-blind, placebo-controlled study that will evaluate the potential of tildacerfont to reduce GC burden in adult subjects with classic CAH who have LLD ≤ A4 ≤ 2.5x ULN and are on supraphysiologic doses of GC therapy (≥30 mg/day and ≤60 mg/day HCe). This will be the first study of tildacerfont to evaluate GC dose reduction. In addition, this study will characterize clinical outcomes after up to 76 weeks of treatment with tildacerfont. An optional Open-Label Extension Period will provide an open-label treatment with tildacerfont at 200 mg QD for up to 240 weeks.
To find out the effects of tenofovir alafenamide (TAF) on children and adolescents with chronic hepatitis B.
Intervention Study for Type 2 Diabetes.
To evaluate the efficacy of tofacitinib based on remission in pediatric participants with moderately to severely active Ulcerative Colitis (UC).
