Prader-Willi Syndrome (PWS)
This research is being done to test the safety and effectiveness of intranasal carbetocin to treat Prader Willi Syndrome.
This research is being done to test the safety and effectiveness of intranasal carbetocin to treat Prader Willi Syndrome.
To demonstrate that an ultra-fast-acting formulation of insulin lispro is non-inferior to Humalog on measures of glycemic control when administered 0 to 2 minutes before eating in combination with basal insulin.
To explore the safety and treatment effect of intravenous (IV) Remodulin as add on therapy in neonates with PPHN compared to placebo.
Study early development in infants who have an older sibling with autism.
To better understand the effects of nutrition in early pregnancy on infant growth and development.
The purpose of the study is to learn about how to improve access to care during adolescence/young adulthood and to see if Virtual Team Clinic is an acceptable form of care. Virtual Team Clinic is a new approach to provide care to (middle/high) school aged patients living with Type 1 Diabetes and their families. This new approach consists of Virtual Team Clinic appointments (primarily telemedicine, and/or in person as necessary). The study also incorporates group visits/medical appointments with a multidisciplinary care team (e.g., RD, RN, Social Worker, etc.). Each group will have a special theme and learning experience. You will learn how to better take care of your diabetes, get support from others, and hopefully have fun.
To explore the possibilities of providing weight management services to teenagers through technology.
Carfilzomib (also known as Kyprolis®) is approved to be used in certain adult patients with relapsed or refractory multiple myeloma (myeloma that returns after a successful course of treatment or myeloma that does not respond to treatment). Multiple myeloma is a type of blood cancer. Carfilzomib is not approved to treat ALL. It has not been given in combination with the other drugs used in this study. This study is being done to find out if carfilzomib can be safely given before and during treatment with standard chemotherapy drugs.
The goals of this study are to improve survival rates in children and young adults with relapsed AML through the combination of DEC and VOR.
This study aims to evaluate the side effects (good and bad) of giving Vincristine Suflate Liposome Infection (also known as Marqibo®) - a drug approved for leukemia treatment in adults but that is experimental in children - with other standard chemotherapy drugs and to measure how effective the combination is at treating children and young adults with leukemia that has come back after initial treatment.