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Monogenic Diseases
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Monogenic Diseases trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Monogenic Diseases 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.
This trial aims to evaluate the prevalence of idiopathic short stature among children whose growth is above -2,5SD (AFPA- CRESS/Inserm -CompuGroup Medical 2018…
The purpose of this research is to develop and validate a single gene Non-Invasive Prenatal Test. The development of this investigational single-gene noninvasiv…
Evaluating a novel, allosteric glucokinase activator in monogenic diabetes secondary to inactivating glucokinase mutations: a randomised, cross-over trial
This study will attempt to identify the specific gene (coded in the DNA) and changes (mutations) within that gene that are the cause of monogenic kidney stone d…
This observational study aims to establish a digital registry, screening platform for patients with monogenic diabetes using internet-based and mobile applicati…
This study evaluates the efficacy of sequential transplantation of umbilical cord blood stem cells and islet cells in children with monogenic immunodeficiency t…
Description of the presence of cell-free fetal DNA in maternal plasma allowed the possibility of non-invasive prenatal diagnosis. Whereas detection of paternall…
Alport syndrome (AS) is one of the most common monogenic kidney disorders, oftentimes leading to end-stage kidney disease (ESKD). As AS is caused by variants in…