The purpose of this research study is to evaluate Parent Child Interaction Therapy (PCIT) delivered via telehealth for young children with autism spectrum disorder (ASD) and disruptive behavior problems. Participants will go through a screening to determine eligibility. Eligible families who choose to participate will complete 3 in-person visits at MUSC and may receive 10 telehealth therapy sessions, at no cost. Families will be compensated for their time.

This observational, multi-center cohort study of pediatric cardiac arrest management will contribute to a clinical CPR Learning Laboratory. The objectives of this study are to characterize the quality of CPR and post-cardiac arrest care delivered to children across a broad spectrum of hospitals, to determine the association between quantitative CPR quality measures (depth, rate, compression release, flow fraction) and survival to hospital discharge, and to determine the association of survival with site-specific post-cardiac arrest care (PCAC). The study will enroll pediatric cardiac arrests requiring chest compressions for ≥1 minute identified as part of standard clinical operations. The CPR quantitative measures, defibrillator data (when available), monitor data (when available), and post-arrest care will be de-identified and submitted to a central database.

This study is for patients with recurrent/progressive medulloblastoma, which is a type of childhood brain tumor. Participants in this study will receive intravenous (IV, into the veins) bevacizumab and intrathecal (into the spinal fluid) or intraventricular (into the fluid surrounding the brain) etoposide and cytarabine in combination with five oral (taken by mouth) chemotherapy drugs as a possible treatment for recurrent/progressive medulloblastoma. Total study duration is about 1 year and depending on how well a participant tolerates the medications and the response of the disease, the patient may continue the treatment after the first year.

Alzheimer's disease and Epilepsy may affect over 80% of individuals that have Down syndrome by the age of 60. Biomarkers found in the blood can enhance our understanding of the earliest changes linked to disease and may enhance clinical detection and healthy aging for individuals with Down syndrome.

The purpose of this study is to discover early neurobiological processes underlying the transition from healthy aging to disease. Our research team has developed technology that allows detection of small changes in the brain that get transferred to the blood.

We are recruiting individuals that either have or do not have Down syndrome for this biomarker study. Participants should be between the ages of 6 months and 85 years old and may include mothers and siblings of a child with Down syndrome. Infants and children will require consent form a parental or legal guardian.

Each participant will provide a blood sample for research purposes. We will also gather some basic health information about senses, habits, exercise level and smoking/vaping exposures.

Babies that are born extremely prematurely are at higher risk of developing chronic (long term) lung disease (CLD) and other complications (problems). The purpose of this study is to test the safety and effectiveness of an investigational drug called mecasermin rinfabate (rhIGF-1/rhIGTBP-3) or SHP607. The researchers want to find out if SHP607 can help reduce the risk of chronic lung disease in babies born prematurely and if it can help reduce the risk of other complications.

The aim of this multi-site study is to evaluate the effectiveness of mHealth intervention strategies for improving anti-epileptic drug adherence in caregivers of young children with epilepsy. A 2-month baseline period will be followed by two stages. In Stage 1 (3-months), caregivers will received either 1) a mHealth education module and automated digital reminders or 2) the mHealth education module, automated digital reminders, and individualized adherence feedback based. In Stage 2 (2-months), caregivers will either receive 1) continued individualized adherence feedback or 2) individualized adherence feedback and a mHealth problem-solving module. The primary outcome is electronically-monitored adherence. Secondary outcomes include seizure severity/frequency, quality of life and healthcare utilization.

Early intervention for infants and toddlers with or at-risk for autism spectrum disorder can promote developmental skills and improve lifelong outcomes. Yet, many children with ASD are not diagnosed until after age 3. In order to improve early detection of ASD, we are investigating very early predictors of social communication challenges in infants as young as 1 week to 6 months of age.

This research study examines how the development of attention and motor skills in the first year of life is associated with the emergence of social and communication skills in three groups of infants: infants who are first born or who have a sibling with no developmental delays, infants who have an older sibling diagnosed with autism spectrum disorder, and infants who were born preterm.

Transforming health care and outcomes for children with rare diseases is difficult within the current health care system. There is great variation in care delivery, inadequate and slow application of existing evidence, and ineffective use of available data to generate new knowledge. Individual care centers have inadequate numbers of patients for robust learning and improvement. In order to redesign the system, changes must take place at multiple levels, including the patient and family, clinician, practice and the network. The purpose of this project is to design, develop, and test further refinements to an improvement and research network focused on HLHS, the most severe congenital heart defect, and to use a registry to simultaneously improve clinical care, redesign care delivery systems and to conduct quality improvement, health services, outcomes, and comparative effectiveness research. The purpose of this initiative, specifically, is to improve care and outcomes for infants with HLHS by: 1) expanding the established NPC-QIC national registry to gather clinical care process, outcome, and developmental data on infants with HLHS between diagnosis and 12 months of age, 2) improving implementation of consensus standards, tested by teams, into everyday practice across pediatric cardiology centers, and 3) engaging parents as partners in improving care and outcomes. We utilize a quality improvement methodology, known as the adapted learning collaborative model, which expedites the implementation of tools and strategies that facilitate changes such as systematic care coordination, cardiovascular monitoring, and nutritional monitoring into every day practice. The NPC-QIC registry is used to document the impact of these changes on various care processes and outcomes (e.g., mortality rate, readmissions, and weight gain).

Adult and Pediatric patients with glomerular disease:
Patients of all ages are needed to participate in a research study to investigate glomerular disease and create a worldwide database to help in the research and future treatment of this disease. To join this study, you must have a type of glomerular disease, have had a first kidney biopsy within the last 5 years, not be on dialysis, or not have had a kidney transplant.

The study is projected to last 4 years, and all study procedures will be done at regular clinic visits (between 1 and 3 yearly). Study procedures include filling out questionnaires and a blood draw at each visit.

Compensation is available for study participation.

This study if for patients that have a blood disease and it's been determined that the best option for treating that blood disease is a cord blood transplant. Cord blood (CB) is blood that is taken from the umbilical cord and placenta of healthy newborn babies after childbirth. The cord blood collected from a newborn baby is called a cord blood unit. The United States Food and Drug Administration (FDA) considers cord blood to be a biological drug. These are considered "investigational" products. This study will evaluate the safety of administration of the investigational cord blood units by carefully documenting all infusion-related problems.