Enhancing NMDA Receptor Signaling to Treat Fragile X Syndrome
FRAXA-backed work revealed NMDA receptors may hold the key to correcting brain signaling in Fragile X, pointing to new treatment strategies.
FRAXA Biotech Games, the Beginning of Something Great
The first FRAXA Biotech Games united 42 Boston-area teams for friendly competition and fundraising to advance Fragile X research.
Federal Funding for Fragile X Research Faces Snags
Federal support for Fragile X research is declining. NIH and the Department of Defense are reducing investment, making advocacy more important than ever.
One Person Can Make a Difference
Elle Skala launched a $10K birthday fundraiser for FRAXA, promising to shave her head if she reached her goal! What an inspiration!
Ganaxolone Fragile X Clinical Trial Showed Disappointing Results
Ganaxolone, an experimental drug from Marinus Pharmaceuticals which targets GABA receptors, did not show promise for Fragile X syndrome in a clinical trial.
FXS Patients’ Social Deficits are Linked to Social Anxiety, Eye-tracking Study Says
Dr. Craig Erickson and colleagues at the University of Cincinnati used eye-tracking technology to understand sociability in Fragile X syndrome. This study affirms what so many parents, caretakers, and educators suspect: people with fragile X want to be social, and it is anxiety – not lack of interest – which usually hold them back. If anxiety could be reduced, more sociability would likely follow. Dr. Erickson is a Fragile X expert and FRAXA investigator who is currently conducting a Fragile X clinical trial of an investigational new drug.
Fragile X Research has More Runners in the Race
We asked for your help to get more runners in the race to find effective treatments and a cure for Fragile X, and you came through for us. Thank you all so much! FRAXA’s 2018 year-end annual appeal raised $733,017 to fund new Fragile X research!
Screening 2,320 FDA-Approved Drugs for Potential Treatment of Fragile X
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.
Kathan Pierce Honored as a New England Patriots Difference Maker
FRAXA Board Member Kathan Pierce was honored by the Patriots & Robert Kraft for her volunteerism. The team also awarded FRAXA a $5K grant!
Novel Modulators of Potassium Channels to Treat Fragile X
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.
Tetra Announces $40M to Advance BPN14770 for FXS and Alzheimer’s Disease
Tetra Discovery Partners has signed a multi-part deal that could bring it up to $160 million, plus royalties, from Shionogi & Co, Ltd, a Japanese major research-driven pharmaceutical company. Tetra currently is conducting an investigational Phase 2 study of BPN14770 in adults with Fragile X Syndrome, an indication for which BPN14770 has received Orphan Drug Designation from the US Food and Drug Administration. This clinical trial was made possible by early work with the FRAXA-DVI and over $200,000 from FRAXA.
Meet Charlie!
Meet #FriendofFRAXA Charlie! If you would like to nominate someone as a #FriendofFRAXA, we welcome all who have been touched by Fragile X, including friends, grandparents, siblings, professionals and companions alike to become a #FriendofFRAXA with the goal of putting a face to Fragile X for those who may not know someone directly.
Hope for the Holidays Gala Helps Advance Fragile X Research
The sold-out Hope for the Holidays Gala raised $100K for FRAXA! Huge thanks to the Pierces for their continued generosity and support.
Coffee, Tea, and Chocolate: Adenosine Receptors in Fragile X
Could “caffeine-like” drugs help Fragile X? FRAXA funded research to test adenosine blockers, which may boost thinking and improve symptoms in Fragile X mice.
Boys Basketball Fundraiser Kicks Off The Season of Giving
60 players and families joined Newton’s 3rd Annual 3-on-3 Basketball Tournament, a fun NAA event full of teamwork and community spirit.
Contacting Your Member of Congress to Advocate for Fragile X Research
A quick guide to contacting Congress for Fragile X advocacy—includes tips, recommendations, and how to look up your senators and representatives.
#GivingTuesday 2018 – Thanks to you!
You did it! The Fragile X community raised over $50K on #GivingTuesday—matched by our Board! Thank you for fueling our annual appeal.
Meet Emily!
Meet #FriendofFRAXA Emily! If you would like to nominate someone as a #FriendofFRAXA, we welcome all who have been touched by Fragile X, including friends, grandparents, siblings, professionals and companions alike to become a #FriendofFRAXA with the goal of putting a face to Fragile X for those who may not know someone directly.
Drug Duo Delivers Brain, Behavioral Benefits for Fragile X Syndrome
Administering a cholesterol drug alongside an antibiotic eases atypical behavior and restores the signaling balance in the brains of people with fragile X syndrome.
Finding Fragile X Biomarkers – From Transcriptomics to Behavior in Patients
FRAXA funded a study using blood-based transcriptomics to find reliable Fragile X biomarkers. This unique approach links molecular data to behavior for future trials.
Research Points to Drugs which Inhibit PDE to Treat Fragile X
FRAXA-funded work identified PDE enzymes as key targets in Fragile X, showing that PDE inhibitors can fix signaling and boost synaptic function. PDE4D trials are underway.
Metformin and Aberrant Insulin Signaling in a Fragile X Mouse Model
FRAXA-funded research is revealing how insulin signaling is altered in Fragile X and whether lowering it, including with metformin, could ease symptoms.
Fragile X Brothers Two of the Pittsburgh Synagogue Massacre Victims
We mourn Cecil & David Rosenthal, beloved brothers with Fragile X, lost in the Tree of Life tragedy. Their kindness and faith will never be forgotten.
Meet Andy!
Meet #FriendofFRAXA Andy! If you would like to nominate someone as a #FriendofFRAXA, we welcome all who have been touched by Fragile X, including friends, grandparents, siblings, professionals and companions alike to become a #FriendofFRAXA with the goal of putting a face to Fragile X for those who may not know someone directly.