The FRAXA Drug Validation Initiative (FRAXA-DVI) provides speedy, cost-effective, objective testing of potential new Fragile X treatments.

Pharmaceutical companies developing medicines for brain disorders like Alzheimers, epilepsy, or depression sometimes find evidence suggesting that their compounds could treat Fragile X. We can arrange to send promising compounds to FRAXA-DVI for further study. It is also a next step for testing new leads from university-based research projects.

Since 2011, Patricia Cogram, PhD, and Dr. Robert M. J. Deacon, PhD, have worked to validate new pharmaceutical leads for the treatment of Fragile X.
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Finding Fragile X Biomarkers – From Transcriptomics to Behavior in Patients

With an award from FRAXA Research Foundation, Dr. Vanderklish (Scripps Institute, USA), Dr Paulina Carulo (FLENI Institute, Argentina) and Dr. Patricia Cogram (GeN, UK) are analyzing patterns in gene expression in blood cells of patients with Fragile X syndrome. They are using “transcriptomics” which can produce a time-sensitive signature of an individual person. This is the first time that all these different levels of study –from transcriptomics to behavior– have been done for individual patients with Fragile X.

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