The goal of this study is to determine whether JASPER (Joint Attention, Symbolic Play, Engagement, Regulation), which is an intensive, targeted early behavioral intervention focused on a ...
Infants with congenital heart disease often require an intervention during their first year of life. Infants are generally admitted to a cardiac intensive care unit and are routinely prescribed stress ...
The objective of the Pediatric Gastroparesis Registry is to create a national prospective registry of children and adolescents with gastroparesis and gastroparesis-like syndrome (symptoms of ...
This study will evaluate the pharmacokinetics (PK), safety, tolerability, efficacy, and pharmacodynamic effect of VX-445, tezacaftor (TEZ), and ivacaftor (IVA) when dosed in triple combination (TC) in ...
The Goal of this study is to investigate if individuals ages 12 years and older, carrying the IL-4RαR576 gene variant, will have a greater response to therapy acting directly on the anti-IL-4R. This ...
The purpose of this study is to investigate the effect of soticlestat on the frequency of motor seizures for participants with Dup15q or CDD during the Maintenance Period.
The PolarisDMD study is a Phase 3, global study to evaluate the efficacy and safety of edasalonexent in pediatric patients with a genetically confirmed diagnosis of DMD. Male patients from 4-7 years ...
Despite major technological advances, management of type one diabetes mellitus (T1D) remains suboptimal, putting millions of people at risk for immediate and long-term complications. After meals, a ...
The main goal of this study is to identify abnormal functional and anatomical brain reorganization associated with hand, foot, and shoulder function in children (0-18 years old) with cerebral palsy ...
