Today, Tetra Therapeutics announces the first unequivocally positive phase 2 clinical trial in Fragile X syndrome, press release below. The results do not depend on carving out a subset of patients or post hoc analysisAn analysis after seeing the test results, and it can lead to bias, because researchers are able to test the data in any way that produces a favorable result. This trial is the result of many years of rational drug discovery funded by FRAXA. The therapeutic target, PDE4, has been identified by multiple researchers including FRAXA Investigators Tom Jongens, Sean McBride, Elizabeth Berry-Kravis, Nobel laureate and FRAXA scientific advisor Eric Kandel. PDE4 is one of the best validated Fragile X treatment targets and we believe this is a genuinely disease-modifying therapy. (Because PDE4 inhibitors are famous for causing nausea; Tetra's compound targets a subtype of PDE4D, thus largely avoiding GI upset.) Many researchers have expressed doubtsRead more
FRAXA Biotech Games™, a gathering of Boston area biotech companies to network in a friendly setting, and form new relationships and potential collaborations while raising money for Fragile X research, is adapting its 2020 event to maintain its commitment to Fragile X and autism research while prioritizing the health and safety of its contestants and volunteers. The organization’s traditional annual event will be transformed into an online interactive experience September 9-11, 2020.
Newburyport, MA (July 15, 2019) — Today, FRAXA Research Foundation announces that 20 buildings, bridges and landmarks across the United States and Canada will be illuminated teal on Monday, July 22, to celebrate International Fragile X Awareness Day. The goal of the initiative is to raise awareness of Fragile X syndrome, the most common inherited cause of autism and learning disabilities worldwide.