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Child Syndrome
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Child Syndrome trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Child Syndrome 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…
ENDEAVOR is a Phase 1/2, 2-part, multicenter study to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet syndrome aged ≥6 to…
.Neurofibromatosis type 2 is an inherently autosomal dominant genetic disease, but cases of mosaicism or de novo mutation are not uncommon. the prevalence is es…
The purpose of this study is to evaluate the efficacy and safety of ION582 in children and adults with Angelman syndrome caused by a deletion or mutation of the…
This basket trial will enroll prepubertal children and adolescents with clinically diagnosed and genetically confirmed (if applicable) TS, SHOX-D, SGA, or ISS b…
Idiopathic Nephrotic Syndrome (INS) is a kidney disease characterized by massive proteinuria and hypoalbuminemia. It includes two anatomopathological entities:…
This phase III trial compares hematopoietic (stem) cell transplantation (HCT) using mismatched related donors (haploidentical \[haplo\]) versus matched unrelate…
Neuropsychiatric impairments in young adults are common and can involve social withdrawal, and difficulties in receiving support from healthcare and municipal s…