This is a Phase 3, global, double-blind, randomized, placebo-controlled study of adjunctive GNX treatment in children and adults with TSC-related epilepsy. The study consists of a 4-week prospective ...
The aim of this study is to test the effectiveness of a single-session, digital intervention teaching the principle of practicing the opposite, when administered to youths on the waitlist for ...
Study the mechanisms underlying action recognition and execution
The purpose of this study is to establish a registry of individuals with confirmed or suspected Chopra-Amiel-Gordon Syndrome (CAGS) to learn more about the range of symptoms, changes in the structure ...
Pathogenic variants in the Cyclin-dependent kinase like 5 (CDKL5) gene cause CDKL5 deficiency disorder (CDD, MIM 300672, 105830), a severe developmental and epileptic encephalopathy associated with ...
This research study is studying a new drug, uproleselan, to see if it is safe and effective in decreasing relapse after stem cell transplant and improving leukemia-free survival in pediatric patients ...
The goals of this study are to 1) pilot the feasibility of a novel meal kit delivery intervention in families and children with food insecurity and obesity and 2) evaluate the implementation of the ...
This study will evaluate the effects of whole vs. nonfat milk consumption on body composition, cardiometabolic disease risk factors, and dietary quality.
Severe Aplastic Anemia (SAA) is a rare condition in which the body stops producing enough new blood cells. SAA can be cured with immune suppressive therapy or a bone marrow transplant. Regular ...
The purpose of this study is to evaluate the device performance and monitor the safety and effectiveness of the Berlin Heart EXCOR Active Driving System while being used with the approved EXCOR ...