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Newborn, Infant, Disease
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Newborn, Infant, Disease trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Newborn, Infant, Disease trials may be worth asking aboutNorth America
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This is a Phase I/II non-randomized clinical trial of ex vivo hematopoietic stem cell (HSC) gene transfer treatment for X-linked severe combined immunodeficienc…
In this study, ANG003, a pancreatic enzyme replacement therapy (PERT; commonly called "enzymes"), is being investigated as a potential treatment for exocrine pa…
The purpose of this study is to evaluate pregnancy-related health outcomes in participants who are exposed to Epidiolex/Epidyolex during pregnancy and their inf…
GB221 is a gene therapy that delivers a working SMN1 gene to the motor neurons of people with spinal muscular atrophy (SMA) Type 1. This study will evaluate the…
The purpose of this study is to determine if postpyloric feedings effectively improve objective measures of pulmonary health in preterm infants with chronic lun…
The purpose of the study is to determine if babies with left or right-sided CDH that undergo the FETO procedure survive more often and have fewer long-term comp…
Unrecognized coarctation of the aorta (CoA) is a life-threatening component of congenital heart disease (CHD) in which narrowing of the aorta causes obstructed…
Background: Clinical Genetics Branch (CGB) researchers study individuals and populations at high genetic risk of cancer in order to improve our understanding o…