Genetics of Autism and other Neurodevelopmental Disorders (NDDs)
To better understand the genetic changes that make children susceptible to the development of autism spectrum disorder, and to study how these changes influence clinical outcomes.
To better understand the genetic changes that make children susceptible to the development of autism spectrum disorder, and to study how these changes influence clinical outcomes.
To investigate the connection between diet, genetics, and diseases in Hispanic / Latino youth.
The collection of the research data we hope will help better screening, diagnosing procedures and treatment of brain injury in newborns and identify a connection between MR imaging and neurodevelopmental outcomes.
To test a new experimental drug Temisirolimus - Temsirolimus (also known as Torisel®) is approved for kidney cell cancer treatment in adults - in combination with approved chemotherapy drugs - in the hopes of finding a drug combination that may be effective against leukemia and non-hodgkin’s lymphoma that has come back after initial treatment. To find the highest dose that can be given without casing severe side effects.
This research is being done to test the safety and effectiveness of intranasal carbetocin to treat Prader Willi Syndrome.
To explore the safety and treatment effect of intravenous (IV) Remodulin as add on therapy in neonates with PPHN compared to placebo.
To determine the safety and feasibility of autologous umbilical cord blood-derived mononuclear cells delivered into the myocardium of the right ventricle during planned Stage II Glen surgical procedure for individuals with hypoplastic left heart syndrome.
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.