Autifony Therapeutics Archives • FRAXA Research Foundation

Neurodevelopmental Drug Development Summit Presentation

January 7, 2022

FRAXA president and co-founder, Katie Clapp was one of three patient advocacy leaders invited to kick off the Neurodevelopmental Drug Development Summit with a presentation on Fragile X, and FRAXA Scientific Advisor, Dr. Elizabeth Berry-Kravis also presented lessons learned from clinical trials in Fragile X Syndrome.

Fragile X Syndrome: In Pursuit of a Cure Webinar

July 29, 2021

Global webinar “Fragile X Syndrome: In Pursuit of a Cure” on July 22, 2021 commemorated World Fragile X Day. Over 5,000 registered from more than 50 countries.

Nazim Kourdougli and Carlos Portera-Cailleau

Correcting Sensory Processing in Fragile X Mice by Modulating Kv3.1

June 19, 2019

FRAXA funded UCLA research on a Kv3.1-targeting drug to ease sensory issues in Fragile X. This work built on Yale-led work now also being pursued by Autifony Therapeutics.

Novel Modulators of Potassium Channels to Treat Fragile X

January 2, 2019

FRAXA-funded Yale research showed disrupted Kv3.1 and Slack potassium channels impair neuronal timing in Fragile X. Published findings support Kv3.1 as a treatment target.

Meng Li, PhD, Xinyu Zhao, PhD, and Anita Bhattacharyya, PhD

FRAXA Research Grants Drive Big Investments in Fragile X

March 28, 2018

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.

Newly Discovered Regulatory Pathways in Fragile X

October 31, 2017

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

August 17, 2017

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.