Holly Roos

Reactivating the Fragile X Gene in Young Mice Reverses Symptoms

Holly Roos Ethell, Iryna • Nelson, David • Razak, Khaleel • Research Updates December 28, 2021January 11, 2022autism, CRISPR, sound

A new FRAXA-funded research project offers hope that Fragile X syndrome could be treated by reactivating the gene which is shut down in people with the syndrome. Researchers at the University of California, Riverside report that they were able to reduce FXS symptoms by inserting the FMR1 gene into the brains of very young mice.

New Fragile X Clinical Trial Announced by Healx

Holly Roos Healx • Research Updates October 21, 2021November 22, 2021drug combinations, drug repurposing, FDA, FRAXA-DVI, Healx

Healx’s AI-driven approach makes finding the right combination therapies more efficient, cost-effective, and rapidly ready for testing at FRAXA-DVI. It was this successful process that has brought Healx to its recent announcement sharing that it has received Investigational New Drug (IND) approval from the US Food and Drug Administration (FDA) for the Phase 2a clinical study of HLX-0201.

Correcting the Brain’s Emotional Memory Center

Holly Roos Chattarji, Sumantra • Research Updates October 19, 2021February 2, 2023amygdala, mGluR, mGluR5, serotonin

Cell Reports has published the results of the study “Correction of Amygdalar Dysfunction in Rat Model of Fragile X Syndrome” by FRAXA Investigator Dr. Sumatra Chattarji and his team at the National Centre for Biological Sciences in Bangalore, India. The researchers investigated the synaptic basis of deficient conditioned fear and its reversal in Fragile X syndrome rats.

Brain Organoids, Moving Fragile X Research Forward

Holly Roos Jin, Peng • Kang, Yunhee • Research Updates • Wen, Zhexing September 9, 2021November 22, 2021Alzheimer's, organoid

There are many ways research produces discoveries, and all of them include a process of steps that build on each other. When an exciting new avenue appeared with potential for Fragile X syndrome, FRAXA stepped up to fund it. We now see the results of this grant and are excited to share them with you. The importance of different types of models have been shared and discussed over many years. We are now adding a “brain organoid” model to this group, and the potential behind it is really exciting.

Promising Results of Preclinical Study of ANAVEX®2-73

Holly Roos Research Updates August 26, 2021August 26, 2021Alzheimer's, autism, BDNF, cancer, FRAXA-DVI, Rett syndrome, seizure

We are excited to share that Anavex Life Sciences announced today that preclinical data of the ANAVEX®2-73 (blarcamesine) study in Fragile X syndrome were published in the peer-reviewed journal, Scientific Reports.

Synaptogenix Announced Intention to Launch a Fragile X Clinical Trial with Bryostatin

Holly Roos Research Updates August 6, 2021November 22, 2021Alzheimer's, autism, FDA, FRAXA-DVI

One of the most exciting kinds of work that FRAXA does is following the journey of an experimental new treatment until it is ready for trials in people with Fragile X. From an initial idea, through the development process, to clinical trials, FRAXA helps out all along the way. From an initial idea, through the development process, to clinical trials, FRAXA helps out all along the way. The recent announcement by Synaptogenix is a great example of how FRAXA funding and use of FRAXA-DVI can accelerate research on Fragile X.