This will be a single-center, cross-sectional survey based study to be distributed to parent/legal guardians of pediatric patients with Celiac Disease during scheduled Pediatric Gastroenterology clinic visits. Eligible participants will complete a single survey regarding their experiences with accidental gluten exposure in their child, associated symptoms, impact on daily activities, and strategies used to manage symptoms. For pediatric patients over the age of 12 and able, will also recommend their assent and participation in survey completion with their parent/legal guardian. Due to infrequency of visits, the survey may also be distributed via a secondary electronic method such as MyChart or email. The aim of this study will be to show among pediatric patients with Celiac Disease, the majority of patients and parents rely on non–evidence-based interventions rather than consulting with a medical provider for symptom control after accidental gluten exposure.

This research study aims to better understand patient experiences with accessing genetic counseling services for hypermobile Ehlers-Danlos syndrome (hEDS). The study will include a review of MUSC medical records to better understand referral patterns to genetic counseling services among patients with hEDS. Patients with hEDS will also be asked to complete a survey about their experiences with accessing genetic counseling services. Healthcare providers will be asked to complete a separate survey about their experiences referring patients with hEDS to genetic counseling services and potential factors that may influence the referral process. The surveys will take approximately 10-15 minutes to complete.

There are minimal risks to participants but may include possible loss of confidentiality or discomfort when answering questions related to healthcare experiences. There is no direct benefit to participants, but the information provided may help improve access to genetic counseling services for future patients with hEDS. Participation in the surveys is completely voluntary.

This study aims to learn how adolescents and young adults who received a liver transplant as children think about their transplant and prepare to manage their healthcare as they get older. Participants will complete questionnaires and may take part in an interview about their experiences, feelings, and healthcare responsibilities. The study will explore how young people view their transplant, how it fits into their sense of self, and how these factors relate to their readiness for adult healthcare. Information from this study will help researchers better understand the needs of young liver transplant recipients and guide the development of future support programs.

This study is recruiting pediatric patients who have been diagnosed with Favorable Histology Wilms Tumor (FHWT). Wilms Tumor is the most common type of kidney cancer in children, and FHWT is the most common subtype. The goal of the study to improve outcomes within cohorts of FHWT patients with current long-term EFS rates at or below 80% and maintain outcomes despite therapy reduction within cohorts of FHWT patients with current long-term EFS rates at or above 84%. Common side effects of treatment in regards to this study include nausea, vomiting, hair loss, and fatigue (tiredness). Participants will receive the medications through an IV in their arm and orally. Study participation is expected to be approximately 5 years.

The FIORELLO study is for pediatric patients who 1) Have heart failure and left ventricular systolic dysfunction associated with dilated cardiomyopathy or congenital heart disease and 2) Have successfully completed the FIORE trial. Study participants will receive the study drug finerenone, which has not yet been FDA approved for this specific pediatric population. The study will last 9 months and the objective is to show that finerenone in addition to standard of care is safe when given long-term to pediatric patients with left ventricular systolic dysfunction.

This is a randomized, double-blind, placebo-controlled, multicenter, parallel-group
study of the efficacy and safety of 2 weight-based treatment groups of plecanatide
versus placebo in children and adolescent participants 6 to less than 18 years of age with FC. Randomization will be stratified by sex to ensure balance across treatment groups.

Approximately 30 participants will be randomized to each of the Placebo, Low
Dose, and High Dose groups, within age group. Low Dose and High Dose will be
assigned once daily treatments based on the weight of the participant at
randomization to achieve weight-based dosing of 0.025 to 0.05 mg/kg in the Low
Dose treatment group and 0.05 to 0.1 mg/kg in the High Dose treatment group.

A germ called Streptococcus pneumoniae (also known as S pneumoniae) is a leading cause of serious diseases including infection of the lungs (pneumonia), infection of the brain lining (meningitis), infection of the blood (bacteremia) and infection of the ears (otitis). There are more than 90 different types of S pneumoniae, some of which cause more serious infections than others. 20-valent (meaning the vaccine protects against 20 different types of S. pneumoniae) pneumococcal conjugate vaccine (Prevnar 20®; 20vPnC) was approved in 2021 first in adults then in children in the United States in 2023, to prevent pneumococcal disease.

The study vaccine, 25-valent pneumococcal conjugate vaccine (25vPnC, PG4 vaccine, pneumococcal fourth-generation vaccine) contains the same components found in 20vPnC, one of which has been changed, but with 5 additional components to help expand protection against 25 types of S pneumoniae. The study vaccine is not approved by the United States Food and Drug Administration (FDA) or elsewhere around the world.

This study will look at how a new pneumococcal vaccine will work in healthy infants while expanding protection against pneumococcal disease due to the germ called S pneumoniae; and, how the new study pneumococcal vaccine will work together with recommended childhood shots.

This study will last for about 16 to 19 months and will have at least 6 visits and 1 telephone contact, about 6 months after your child's last study vaccination.

This study is recruiting patients receiving cancer treatment that includes a type of drug called an "alkylating agent", which may cause side effects such as damage to the ovaries (female reproductive organs). The goal of the study is to determine the feasibility of conducting a cross network, multi-site, randomized clinical trial of triptorelin among newly diagnosed adolescent and young adult (AYA) female cancer patients age <40 years (exclusive of breast cancer). The triptorelin used in this study may affect how different parts of your body work such as your liver, kidneys, heart, and blood. Participants will receive the medications through an intramuscular (IM) injection in their arm. Study participation is expected to last up to three years.

This is a 3 month study for pediatric patients between the ages of 6 months and 18 years that have a current diagnosis of heart failure. This study will compare the use of the medication finerenone to placebo. During the study, participants will continue to receive their standard of care therapy according to pediatric heart failure guidelines. At the end of the 3 month study period, participants will have the option to enroll in a 9 month open label extension.

The purpose of this research is to assess a stepped care model of a telehealth parenting program for children ages 2-6 years old with a neurodevelopmental diagnosis and behavior problems. Families will complete an online intake assessment, 6 group-based tele-health Parent-Child Interaction Therapy (PCIT) sessions, and an online post intervention assessment. Some families may receive additional individual virtual booster sessions and will complete a second online post assessment.