Autifony Therapeutics Archives • Fragile X Research

FRAXA Research Grants Drive Big Investments in Fragile X

Dave Bjork Bhattacharyya, Anita • Canal, Clinton E. • Kaczmarek, Leonard • Research Updates • Zhao, Xinyu March 28, 2018July 10, 2018Autifony Therapeutics, FRAXA-DVI, Fulcrum Therapeutics, serotonin, sound

Most people know that FRAXA supports academic research at many institutions such as Harvard University, University of Pennsylvania, Massachusetts Institute of Technology, and Yale University. However, FRAXA is also working with more than 30 pharmaceutical companies around the world. Mike spends a lot of his time advising and collaborating with industry partners.

Novel Modulators of Potassium Channels to Treat Fragile X

FRAXA Research Foundation 2015 Grants • 2016 Grants • 2017 Grants • Disease Mechanisms • Kaczmarek, Leonard • Treatment Targets • Yale University December 25, 2017February 6, 2019Autifony Therapeutics, autism, glutamate, hypersensitivity, ion channel, mGluR, mGluR5, mRNAs, potassium channels, sound

With funding from FRAXA over 2015-2017, the Yale University team of Leonard Kaczmarek, PhD showed that the firing patterns of auditory neurons in response to repeated stimulation is severely abnormal in Fragile X mice. Based on these results, they are collaborating with the UK-based company Autifony to develop advanced compounds which may reverse these deficits.

Newly Discovered Regulatory Pathways in Fragile X

J. Dora Levin Kaczmarek, Leonard • Research Updates October 31, 2017July 30, 2018Autifony Therapeutics, BK channel, FDA, glutamate, hyperexcitability, hypersensitivity, mGluR5, potassium channels, seizure

Studies at Yale University and elsewhere are showing that FMRP plays a significant role in the regulation of potassium channels. Looking forward, potassium channel opener drugs could rescue some symptoms of Fragile X in humans.

Neural Markers of Fragile X: A Powerful New Tool for Clinical Trials

J. Dora Levin Biomarkers • Boston Children's Hospital • Clinical • Harvard University • Nelson, Charles A. • Research Updates • Wilkinson, Carol August 17, 2017July 30, 2018Autifony Therapeutics, autism, EEG, hypersensitivity, ion channel, Pierce Family Fragile X Foundation, sleep, sound

Once the neural marker is identified for a particular challenge, such as kids with poor language versus good language, neural markers can be measured during drug and behavioral therapy trials to see if a child is improving based on objective biological measures.