Cogram, Patricia Archives • Fragile X Research

Tetra’s Fragile X Clinical Trial – The Most Successful So Far

FRAXA Research Foundation Berry-Kravis, Elizabeth • Cogram, Patricia • Research Updates September 7, 2021November 22, 2021BPN14770, cyclic AMP, drug tolerance, FRAXA-DVI, NIH, PDE4, Tetra Therapeutics

Dr. Mark Gurney, CEO of Tetra Therapeutics, discusses how one of the earliest clues to the biology of Fragile X led to the most successful Fragile X clinical trial to date. FRAXA and Tetra began working together after a key FRAXA-funded study caught the attention of Dr. Gurney. Through the FRAXA Drug Validation Initiative, Dr. Patricia Cogram was able to conduct preclinical validation experiments with Tetra’s lead compound in record time, paving the way for clinical trials.

FRAXA Drug Validation Initiative (FRAXA-DVI)

FRAXA Research Foundation 2011 Grants • 2012 Grants • 2013 Grants • 2014 Grants • 2015 Grants • 2016 Grants • 2017 Grants • 2018 Grants • 2019 Grants • 2020 Grants • 2021 Grants • Biomarkers • Carullo, Paulina • Cogram, Patricia • Current Research Grants • Deacon, Robert • Drug Repurposing • FLENI Foundation • Research Resources • University of Chile • University of Oxford May 6, 2021May 6, 2021BPN14770, drug repurposing, EEG, FRAXA-DVI, Healx, microbiome, Neuren Pharmaceuticals, NIH, Ovid Therapeutics, PDE4, Tetra Therapeutics, trofinetide

The FRAXA Drug Validation Initiative (FRAXA-DVI) provides speedy, cost-effective, objective preclinical testing of potential Fragile X treatments. FRAXA-DVI uses in-vitro systems, behavior batteries, and gene expression and peripheral biomarker platforms to validate investigational new drugs and repurposed available compounds in Fragile X syndrome (FXS).

Finding Fragile X Biomarkers – From Transcriptomics to Behavior in Patients

FRAXA Research Foundation 2018 Grants • Biomarkers • Carullo, Paulina • Cogram, Patricia • Current Research Grants • Scripps Research Institute • Vanderklish, Peter November 6, 2018January 3, 2019EEG, FRAXA-DVI, mRNAs

With this $20,000 award from FRAXA Research Foundation, Dr. Vanderklish and collaborators at Scripps Research Institute, the University of Chile, and the FLENI Institute in Argentina 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.

In Their Own Words: Reports From the International Fragile X Workshop

FRAXA Research Foundation Cogram, Patricia • Contractor, Anis • Events Recap • Kooy, Frank • Lee, Jeannie • McGill University • Moine, Herve • Razak, Khaleel • Research Updates • Smith, Carolyn B. • Sonenberg, Nahum • Todd, Peter • Vanderklish, Peter October 29, 2017December 26, 2019amygdala, autism, bumetanide, DgkK, drug repurposing, drug tolerance, EEG, ERK, FDA, Fragile X Meeting, GABA, ganaxolone, glutamate, hyperexcitability, hypersensitivity, metformin, mGluR5, minocycline, mRNAs, NIH, Rett syndrome, seizure, sleep, sound

The 18th International Fragile X and Related Neurodevelopmental Disorders Workshop in Quebec, Canada, was a great success, featuring Fragile X much more heavily than any previous meeting in this series! We asked our speakers to summarize their work in their own words, with brief updates from researchers investigating Fragile X.

Treatment of Fragile X Syndrome via Dopamine Enhancers and Glutamate Inhibitors

FRAXA Research Foundation 2006-2010 Grants • 2011 Grants • Cogram, Patricia • Cowan, Justin • University of Chile June 2, 2013November 22, 2021dopamine, glutamate

FRAXA Awards $50,000 in 2011 and $50,000 in 2010 to Patricia Cogram, PhD for treatment of Fragile X syndrome via Dopamine Enhancers and Glutamate Inhibitors. This project aims to follow up our and others observations that the dopamine receptor is under expressed in the Fragile X syndrome and thus determine the effectiveness of targeted pharmacological treatments in Fragile X syndrome.