TReatment for ImmUne Mediated PathopHysiology (TRIUMPH) is a multi-center, three-arm clinical trial of immunosuppressive therapy for children with acute liver failure. This is a double-blind study, so the study doctors and participants will not know to which arm the participants are assigned. The study will determine if corticosteroids or equine anti-…
The purpose of this research study is to determine if an investigational drug, VS-01, will help in the treatment of acute-on-chronic liver failure (ACLF) compared to supportive care (standard of therapy). ACLF is a potentially life-threatening condition characterized by a group of signs that occur together (syndrome) in patients with a late stage of…
Down syndrome (DS) occurs in roughly 1 out of 700 births in the U.S. Half of all children with Down syndrome have congenital heart disease (CHD), often requiring corrective heart surgery. Even though CHD in children without DS is known to be associated with developmental problems, few studies have explored how CHD can affect the development and behavior of…
This is a study designed for patients with medullary thyroid cancer. Treatment will consist of a drug called Ponatinib. Ponatinib is an investigational agent that blocks abnormal cancer proteins and therefore harms cancer cells. It was recently approved by the Food and Drug Administration for leukemia treatment, but is not approved for medullary thyroid…