In this research study we want to learn more about using non-invasive tools as a way to predict whether or not a child under general anesthesia will require and respond to fluid administration. It is ...
This is a single-arm, multi-site, single-dose, Phase 1/2 study to assess ST-400 in 6 subjects with transfusion-dependent β-thalassemia (TDT) who are ≥18 and ≤40 years of age. ST-400 is a type of ...
The purpose of this is study is to evaluate the effects of DCCR (diazoxide choline controlled release tablets) in children and adults with Prader-Willi syndrome.
The goal of this study is to identify effective ways to assess the patient and family experience. Specifically, the study will examine automated administration of the Child Hospital Consumer ...
Pediatric chronic pain disorders are common and consequential in Western societies, occurring in 25-80% of population-based samples with a median prevalence of 11-38% and significant pain-related ...
This study will evaluate the long-term safety and tolerability of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous ...
New daily persistent headache (NDPH) is a primary headache disorder characterized by the daily and unremitting headache pain patients experience with a distinct onset. Despite the known significant ...
According to normal physiology, the longer fasting period allows food particles to pass stomach through small intestines to minimize intragastric content. The practice guidelines recommend 2-hour ...
This is a Phase 2 multicenter, double-blind, randomized, placebo-controlled study assessing the efficacy and safety of lenabasum for the treatment of cystic fibrosis in patients 12 years of age or ...
This is a prospective, single center, safety and feasibility trial to evaluate the transplantation of the left atrium and pulmonary veins in patients with pulmonary vein stenosis. Consented patients ...