Scunthorpe General Hospital, United Kingdom

The Sweet Dreams Study - Accuracy of Sleep Trackers in Children

Participants will meet the research team at Arkansas Children's Hospital (ACH) two to three hours before their scheduled sleep test and are required to bring a stool sample from the child to the visit. During the visit, the research team will gather information about the child's home environment and medical history, including parent marital status, parental education level, household income, and other factors. Questionnaires will also cover topics such as the child's growth and development, diet, physical activity, use of substances like tobacco and alcohol, and mood. Any questions that cause discomfort may be skipped by the child or their parent. A brief physical exam will be conducted to measure the child's weight, height, blood pressure, and heart rate. Sleep trackers will then be programmed and placed, with two worn on the wrist and two around the waist. Instructions on how to remove and reattach the trackers will be provided to ensure proper use. After completing the sleep test at ACH, the sleep trackers must be left in the sleep clinic. All four trackers must be returned, and parents will be asked to report if the trackers were removed at any point during the study.

A Phase 2 Study of Mutant-selective PI3Kα Inhibitor, RLY-2608, in Adults and Children With PIK3CA Related Overgrowth Spectrum and Malformations Driven by PIK3CA Mutation

A Study to Investigate LP352 in Children and Adults With Developmental and Epileptic Encephalopathy (DEE)

A Phase 3, Placebo-Controlled Study to Investigate LP352 in Children and Adults With Dravet Syndrome (DS)

A Study to Investigate LP352 in Children and Adults With Developmental and Epileptic Encephalopathies (DEE)

Whey Protein Ingestion and Glucose Control in Pre- and Post Diabetic Individuals

Protein consumption in the morning has been shown to reduce appetite and caloric intake (19). In addition, premeal whey consumption reduces post prandial blood glucose, reduces gastric emptying rate, and increases peak blood insulin (10). Thus, it is proposed that ingestion of whey protein within 1hr of waking (and prior to breakfast) and 30 minutes prior to dinner will be more effective in suppressing appetite, carbohydrate intake, and glucose AUC. Ingestion upon waking will mitigate the cortisol-induced drive for carbohydrate intake. Ingestion prior to dinner, the most frequently consumed and largest meal in America (20), will reduce food intake, post meal blood glucose, and insulin area under the curve (21). Specific Aims 1. Determine the effect of WP ingestion within 1hr of waking and prior to breakfast, and 30min prior to dinner, on appetite suppression, carbohydrate and caloric intake, and 24hr glucose AUC over 7d in pre-diabetic (A1C 5.7%-6.4%) and diabetic (A1C 6.5%-7.5%) individuals. 2. Determine the effect of WP ingestion within 1hr of waking and 30min prior to dinner on changes in OGTT, Matsuda index, and whole-body protein balance (compared to control) before and after 7d of WP consumption in pre-diabetic and diabetic individuals. 3. Determine outcome differences between pre-diabetic and diabetic individuals.

The BEE-Power Study (Boosting Exercise for Excellent Pediatric Blood Pressure)

Our approach to testing our working hypotheses will be to enroll children ages 13 to 17.5 years with confirmed diagnosis of HBP. Children will be divided equally into two groups (aerobic exercise and isometric exercise groups), with 18 children in each group. Children who meet inclusion criteria will be asked to attend three study visits at the Arkansas Children's Nutrition Center (ACNC). Resting and 24-hour ambulatory blood pressure measurements will be conducted twice during the study: baseline measurement at visit 2 and after a single 50-minute session of exercise at visit 3. To accommodate for any potential dropouts post-assignment and to maintain an even distribution of participants between groups (n =18 each), the study will enroll up to n = 45 children (Refer to the Data Analysis and Randomization section for more details). The study will follow a randomized parallel group design and will be conducted by the Physical Activity Core - Arkansas Children's Nutrition Center (ACNC) / Arkansas Children's Research Institute in collaboration with the Hypertension Clinic - Nephrology Division at the Arkansas Children Hospital (ACH). Measurements will be completed within four weeks of enrollment. Research staff involved in study procedures are or will be properly trained. All assessments may be repeated if/when needed, and if the participant is willing. Study visits may last up to 3 hours. Baseline characteristics such as socioeconomic status, pubertal stage, dietary quality, physical fitness (aerobic capacity, body composition) and metabolic phenotyping (glucose, insulin, lipids, etc.) will be used to control for baseline covariates as described in the Data analysis Section.

Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy.

This is a multi-center, observational, prospective, longitudinal registry designed to collect data in male patients aged 2 years and older with DMD treated with AGAMREE®. This registry will be conducted in the US, at approximately 25 sites known to treat and follow patients with DMD. The registry plans to enroll approximately 250 male patients aged 2 years and older with DMD. Evaluations will include: - Growth parameters - Body mass index (BMI) - Vital Signs - Physical Exam - Laboratory (Chemistry and Hematology) - North Star Ambulatory Assessment (NSAA) - Performance of Upper Limb (PUL) - Cardiovascular status - Fractures - Bone density - Puberty - Quality of life (QoL) - Adverse events (AEs) Patients will be followed for approximately 5 years in the registry and will return to the site for Yearly Follow-up Visits (+/- 30 days) for registry assessments. Information on standard of care treatment and procedures for management of DMD will also be collected. Patients and/or their parents/legal guardians will be asked to complete paper QoL questionnaires at enrollment and at each Yearly Follow-up Visit (+/- 30 days).

An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscular Dystrophy

A Study to Assess Adverse Events, Change in Disease Activity, and How the Drug Moves Through the Body in Children With Juvenile Psoriatic Arthritis (jPsA) Receiving Subcutaneously Injected Risankizumab or Adalimumab