Transcriptomic Analysis of Fibroblasts and Blood in Patients With Rare Diseases

Recruiting N/A Interventional Study
Rare Genetic Disease
No Placebo Group Every participant receives an active treatment — no one gets a placebo.
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At a Glance
Age
0 – 99
Sex
Any
Study type
Interventional
Purpose
Diagnostic
Participants needed
62 (estimated)
Sponsor
Assistance Publique Hopitaux De Marseille · Other
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About This Trial
This study aims to answer a key question in the field of rare genetic diseases by determining the prevalence of deleterious variants at RNA level in undiagnosed patients with intellectual disability and/or neonatal hypotonia. This study will put an end to diagnostic erraticism in a number of patients. Finally, the results of this study will make it possible to compare the two types of tissue used…
Trial Locations
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Eligibility Criteria
Inclusion Criteria: * Male or female, aged 0-99 years * Patient with neonatal intellectual disability and/or hypotonia followed at one of three inclusion centers * Patient or parent has been informed about the study and has signed an informed consent form * Genetic analysis by high-throughput DNA s…
Contacts

Svetlana GOROKHOVA, MD

33491388499

svetlana.gorokhova@ap-hm.fr

CONTACT