Early intervention for infants and toddlers with or at an elevated likelihood 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 4 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.

The goal of this project is to better understand the relationship between tobacco/nicotine and cannabis using behavioral economics during a tobacco/nicotine quit attempt. All participants will receive tobacco/nicotine cessation treatment (smoking and/or vaping treatment) for 12 weeks. To qualify, participants must be between the ages of 18-25 and use tobacco products (smoke cigarettes and/or vape nicotine) and use cannabis (in any form). Participants do not need to be interested in quitting cannabis/marijuana to qualify. This study is being conducted by the Medical University of South Carolina. All procedures are conducted remotely and there is no in-person visits are needed.

The purpose of this research is to assess a group-based telehealth parenting program for child ages 2-6 years old with a neurodevelopmental diagnosis and behavior problems. Families will complete an online intake assessment, 6 group-based telehealth PCIT sessions, and an online post intervention assessment. Families will be compensated for their time.

The purpose of this study is to evaluate the safety and effectiveness of acoramidis (AG-10) and determine if it can help people with the genetic TTR variant that can cause ATTR-CM. The sponsor (Eidos) is conducting a research study to see if the study medicine will help in slowing the development and/or progression of a rare disease called Symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM). AG-10 is an investigational drug. "Investigational" means that AG-10 is not yet approved for use in any settings outside of clinical research studies like this one and is considered experimental. Cardiomyopathy is a disease of the heart muscle that makes it harder for the heart to pump blood to the rest of the body. AG-10 has been shown to reduce the level of TTR in the blood of animals and healthy volunteers tested to date. Reducing the amount of TTR in your blood may reduce the amount of amyloid deposits in your body and may keep your cardiomyopathy from getting worse over time.

If you meet the requirements and agree to participate in this study, you will go through a 2 part screening period and if approved you will begin treatment. You will be randomized, like flipping a coin, to drug or placebo and take two pills once every day for 60 months.
Your participation in this study will last up to 60 month and will consist of clinic visits and follow up visits at home via the telephone. Some tests requires of you will include an echo, ECG, cardioradionuclide scans (scans that use radioactive agents to look at images of the heart like a PET scan), MRIs, blood tests, nerve conduction test, urine samples, and skin biopsies.

This study aims to evaluate the effectiveness of ruxolitinib cream compared to a placebo (vehicle) cream in treating vitiligo in children aged 2 to 12 years. The vehicle cream looks identical to the ruxolitinib cream but contains no active medication. By comparing these two treatments, researchers hope to determine whether ruxolitinib is more effective than the placebo in improving facial and body vitiligo symptoms. This study could provide important insights into new treatment options for young children affected by this condition.

The purpose of this research study is to evaluate Parent Child Interaction Therapy (PCIT) delivered via tele-health for young children with disruptive behavior problems who are at risk for life stressors. Participants will go through a screening to determine eligibility. Once screening is complete, eligible families who choose to participate will complete questionnaires, 3 virtual visits, and will receive 10 tele-health therapy sessions, at no cost. Families will be compensated for their time.

El propósito de este estudio de investigación es evaluar la Terapia de la Interacción Padre-Hijo (PCIT, por sus siglas en inglés) mediante telesalud para niños pequeños con dificultades de comportamiento que están en riesgo de factores de estrés en la vida. Los participantes pasarán un cernimiento para determinar su elegibilidad. Una vez que se complete, las familias elegibles que decidan participar completarán cuestionarios, 3 visitas virtuales y recibirán 10 sesiones de terapia por telesalud, que serán gratuitas. Las familias recibirán compensación por su tiempo.

Cancer caregivers in emerging and young adulthood (ages 18-35) are an underresearched and unsupported group of caregivers, and yet they are not uncommon. To address this critical support gap, the goal of this study is to develop and pilot test a caregiving support intervention specifically tailored for emerging and young adults caring for a parent with cancer. This phase of the study is intended to capture feedback on the intervention via focus groups with emerging and young adult caregivers of a parent with cancer.

This study aims to evaluate the remote delivery of self-regulation assessments in young children with autism and their parents. Parents will complete online surveys. Parents and children will complete two virtual visits. At the second visit, participants will wear a heart rate monitor while completing study tasks. This study will inform research on behavioral therapies for children with autism.

Written Exposure Therapy (WET) is a five-session mental health therapy for post-traumatic stress disorder (PTSD). Research shows that it works as well as longer treatments for PTSD among people over 18, even though it requires fewer sessions than other PTSD therapies. However, WET has not been adapted and tested via telehealth with people aged 12 to 18. Our study aims to see how WET can be adapted to meet the needs of people aged 12 to 18 who have experienced trauma and currently have PTSD symptoms. To adapt WET for this age group, first we will talk with PTSD experts and people aged 12 to 18 to learn what changes might make WET more suitable for young people. We'll also deliver WET via telehealth to five people aged 12 to 18 following the manual as it is written for people over age 18 to see what needs adjusting. In the next part of the study, we will offer the adapted version of WET via telehealth to 20 people aged 12 to 18 in our MUSC integrated pediatric primary care clinic. People aged 12 to 18 will take part in five to seven weekly online therapy sessions and three virtual study visits (before therapy, after therapy, and one month later) to better understand who they are as a person and their current mental health symptoms and diagnoses. Individuals who are 18 can participate without caregiver permission; individuals aged 12 to 17 can only participate with caregiver permission. Our goal is to find the best way to provide effective PTSD treatment for young people that can be delivered in real-world pediatric primary care settings, so that ultimately more people can get the help they need after traumatic experiences.