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Congenital Pediatric Disorders
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Congenital Pediatric Disorders trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Congenital Pediatric Disorders trials may be worth asking aboutNorth America
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Trials may have sites in multiple countries. Filtering shows any trial with at least one site in the selected country.
The study will evaluate the tolerability, safety and efficacy of gene therapy product in boys with Duchenne muscular dystrophy (DMD). In Phase I the participant…
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 goal of this observational study is to learn how four acetabular osteotomy techniques change the shape of the hip socket in children and adolescents with de…
This is a Phase 1 study to determine the safety and efficacy of allogeneic neonatal mesenchymal stromal cells (nMSCs) for the treatment of Dilated Cardiomyopath…
SickKids and UHN have developed MedlyPeds which is a smart phone application designed to remotely monitor patients with heart disease. This is a feasibility stu…
An open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of migalastat treatment in pediatric subjects 2 to \< 12…
This is a multi-center, prospective, parallel-group, open-label, randomized clinical study in one hundred and twenty-six (126) neonates and infants diagnosed wi…
A roll-over study to assess long-term effect in pediatric patients treated with dabrafenib and/or trametinib.