How FRAXA Prioritizes Research, Explained
Dr. Mike Tranfaglia explains how FRAXA prioritizes research and the importance of looking at research from multiple angles. "It’s not either-or. It’s not we have a definitive treatment or we have a new drug treatment or we have a repurposing treatment. We can have all of those things, mixed or matched, in a personalized medicine kind of way and I think that’s what we’re headed for."
Aggression, Other Fragile X Behaviors Tend to Ease Over Teenage Years
Behavioral problems such as hyperactivity and aggression are generally more frequent in younger children with Fragile X syndrome, becoming less common as they grow through adolescence and journey toward adulthood, researchers report.
Cape Coral Touch-A-Truck, Free, Family-Friendly Event
Join EHL Fragile X Foundation’s free Touch-A-Truck in Cape Coral! Explore fire trucks, police bikes, ambulances, swamp buggies, and more.
Wine and Friends Are a Great Blend for Fragile X Research
Debra and Kevin hosted their first annual event for FRAXA in honor of their grandson Jackson, who has Fragile X syndrome.
What FRAXA Is Excited about in the Upcoming Fragile X Research Grants, Explained
Dr. Mike Tranfaglia shares what FRAXA is excited about as we work through reviewing all of the submitted Fragile X research grant applications. We find it especially exciting that so many new clinical trials are starting right now, as our major emphasis is getting the drugs and other treatment strategies that we have tested in the Fragile X mouse model to patients in clinical trials.
Gordon Research Conference for Fragile X and Autism-Related Disorders, Explained
The Fragile X and Autism-Related Disorders Gordon Research Conference is a biannual event that provides an international forum for the presentation and discussion of frontier research in these conditions. Dr. Mike Tranfaglia explains why this is the premier conference for researchers and the scientific community.
Making Mittens for Fragile X
Carol spends over 2 hours crafting each fleece-lined mitten. Her handmade creations warm hands and support Fragile X research.
Meet Archie!
Meet #FriendofFRAXA Archie! 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.
Correcting Fragile X Syndrome Deficits by Targeting Neonatal PKCε Signaling in the Brain
Enhancing PKCε in early development normalized oxytocin, AMPAR signaling, and adult behavior in Fragile X mice, highlighting PKCε as a promising therapeutic target.
Climbing 3 Mountains for Fragile X Research
Michael Leonard climbed three mountains in 24 hours to honor his son, Cole, who lives with Fragile X syndrome. A father’s love knows no limits.
Potential Upcoming Advances in Fragile X Research
Dr. Peter Kind, Director of the Patrick Wild Centre and Professor of Developmental Neuroscience at the University of Edinburgh, and Dr. Nahum Sonenberg, James McGill professor of biochemistry at McGill University, share their optimism about the next 10 years of Fragile X research. They discuss where they think the next big discoveries will emerge.
Lovamix: Clinical Trial of Combined Treatment of Minocycline and Lovastatin in Fragile X Syndrome
FRAXA funded the LovaMiX trial of lovastatin + minocycline for Fragile X. 2022 results show safety and support continued study of this dual-target treatment approach.
FRAXA’s Most Successful End-Of-Year Campaign Ever!
Together, we made history! FRAXA’s 2021 campaign was our most successful ever, fueling hope for treatments and a cure for Fragile X.
Neurodevelopmental Drug Development Summit Presentation
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.
Reactivating the Fragile X Gene in Young Mice Reverses Symptoms
Fragile X syndrome might be treated by reactivating the gene which is shut down in the syndrome. Researchers were able to reduce FXS symptoms by inserting the FMR1 gene into the brains of young mice.
Meet Jonathan!
Meet #FriendofFRAXA Jonathan! 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.
10 Year Vision for Collaborations That Transform Fragile X and Autism Research
The future offers hope for people living with Fragile X syndrome. Collaborations between the Fragile X community and other disability organizations help to provide understanding and advancement of research to bring effective treatments to families. FRAXA’s Dr. Mike Tranfaglia talks with Autism Science Foundation’s Allison Singer about the importance of their collaboration as we look forward to the next 10 years.
Patrick’s PALS Still Shines Strong Even in the Face of COVID
Patrick’s PALS 25th Annual Fundraiser raised $140K for FRAXA in 2021, proof that the spirit and generosity behind this event never fade.
16th Annual Fragile X Poker Run Raises nearly $29,000
The 16th Annual Fragile X Poker Run brought friends from 10 states and Israel together, raising $29K for FRAXA research!
GABA-A Receptor in Fragile X Syndrome
Published results from Dr. Frank Kooy reveal how GABAA receptor changes may hold the key to treating Fragile X syndrome.
Characterization of a Novel CYFIP1 – Derived Peptidomimetic Restoring the Dysregulated mRNAs Translation: Toward An Innovative Therapeutic Strategy for FXS
This team is designing tiny “peptidomimetic” drugs that mimic FMRP’s function to rebalance protein production in the brain, aiming to treat Fragile X at its source.
Bruins 50/50 Benefits Fragile X Research
FRAXA volunteers sold 50/50 raffle tickets at the Bruins game, raising $28,040 with the Bruins Foundation, half to FRAXA, half to one lucky fan!
FX-Learn Clinical Trial for Children with Fragile X
Thirteen centers across the US enrolled children with Fragile X in a large-scale clinical trial of Novartis AFQ056. Dr. Elizabeth Berry-Kravis and colleagues aim to show that this targeted treatment — an mGluR5 blocker for Fragile X which failed in previous adult human trials — can be better evaluated by studying effects on learning in young children.
Over $20,000 Raised For Fragile X Research at Callum Cup V
After a year off for COVID, the Callum Cup returned! Millburn FC’s marquee charity match honors Callum Murphy, son of goalkeeper Andrew.