Tetra Therapeutics announces the first unequivocally positive phase 2 clinical trial in Fragile X syndrome. The results do not depend on carving out a subset of patients or post hoc analysis.
This article discusses Abilify (aripiprazole) as a “off-label” treatment for people with Fragile X syndrome (FXS). Abilify targets irritability, aggression, self-injury and severe tantrums.
In this webinar we hear from Alysson R. Muotri, PhD, Professor at University of California San Diego Stem Cell Program and Fabio C. Tucci, PhD, CEO and co-founder at Epigen Biosciences.
Tetra Therapeutics and Shionogi announced plans to expand their partnership supporting BPN14770, a treatment candidate for disorders marked by cognitive and memory deficits.
An obstacle in FXS research has been lack of reliable, unbiased biomarkers to assess herapies. This team found striking similarity in EEG biomarkers between mice and humans.
FRAXA funded a screen of 2,320 FDA-approved compounds in the Fragile X fly model to identify hits that improve memory and social behavior for advanced testing.
Tetra conducted a Phase 2 study of BPN14770 in adults with Fragile X Syndrome. This clinical trial was made possible by early work with the FRAXA-DVI and over $200,000 from FRAXA.
This 2-Period Crossover Study of BPN14770 is accepting adults males with Fragile X syndrome at Rush University Medical Center in Chicago. Principal Investigator of the study is Elizabeth Berry-Kravis, MD, PhD.
A selective inhibitor of the phosphodiesterase type-4D (PDE4D), BPN14770 has shown the ability to improve the quality of connections between neurons and to improve multiple behavioral outcomes in the Fragile X mouse model.
FRAXA partnered with Healx to use AI to find approved drugs and drug combos that could treat Fragile X. Top candidates are now being tested in Fragile X models.
Studies at Yale University and elsewhere show FMRP plays a significant role in regulation of potassium channels. Potassium channel opener drugs could rescue some symptoms of Fragile X.
This work established a high-content synaptic imaging platform for Fragile X cells to test many candidate drugs for their ability to repair synapse structure and function.
Carolyn Beebe Smith studies sleep disruptions in Fragile X and tests whether improving sleep with existing drugs can reduce symptoms and enhance behavior.
In this initial trial with a relatively short treatment period, trofinetide was very well tolerated, with the high dose (70 mg/kg twice daily) demonstrating a consistent pattern of clinical improvement.
With this grant from FRAXA, Dr. Peter Vanderklish explored AMPK activators to treat Fragile X. Both metformin and resveratrol, found in red wine, are AMPK activators.
Wondering which Fragile X trial is right? Eligibility varies, so most families qualify for just one. Talk with your closest clinic to find the best fit.
