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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
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Since the discovery of a small fraction of circulating cell-free fetal DNA (ccffDNA) in the blood of the mother, non-invasive prenatal diagnosis (NIPD) techniqu…
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 is a Phase 2 trial designed to evaluate the clinical efficacy, safety, and tolerability of MAS825 in patients with NLRC4-GOF, XIAP deficiency, or CDC…
Background: The immune system defends the body against disease. It has many different parts spread out in the body, including in the blood and skin. To learn…