The Millburn Football Club in Millburn, New Jersey hosted their 4rd annual Callum Cup. This intra-club charity match (all proceeds being donated to FRAXA) has become the marquee event of the Millburn FC calendar, played in honor of Callum Murphy, the son of club goalkeeper, Andrew Murphy. Callum was diagnosed with Fragile X syndrome when he was 2 years old.
Read more
NH Fragile X Awareness Day Proclaimed at July Jam Fundraiser
On July 22, 2019, the Fragile X July Jam in Derry, NH, raised over $6000 for FRAXA Research Foundation, to fund research to find effective treatments and ultimately a cure for Fragile X syndrome. Governor Chris Sununu’s policy director D.J. Bettencourt read an official proclamation calling for awareness of, and research on, Fragile X.
Read more
20 Landmarks Will Light Up for Fragile X Awareness Day
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.
Read more
Patrick’s PALS 23rd Annual Basketball Tournament Raises $140,000 for Fragile X Research
Patrick’s PALS has grown from a twelve-team tournament to a 36-team extravaganza! On four half-courts, 18 first-round games set teams on a path to the winners or losers bracket. After that, it was all about staying alive and avoiding elimination by not losing two games. In the end, two veteran teams faced off for the rights to ensure their names would be engraved on the PALS Trophy: the multiple-past-champion Kline Team and the always-in-the-mix Gray Team (pictured above). Gray entered the finals with one loss, so they needed to beat Kline twice. That they did to become the Champions of Patrick’s PALS XXIII!
Read more
Correcting Sensory Processing in Fragile X Mice by Modulating Kv3.1
FRAXA awarded a $90,000 grant to Carlos Portera-Cailliau, PhD and Nazim Kourdougli, PhD at UCLA to investigate whether a novel drug can rescue sensory processing deficits in Fragile X mice. People with Fragile X have similar problems in sensory processing. This new drug acts on Kv3.1, a promising Fragile X treatment target also being pursued by UK-based Autifony Therapeutics based on FRAXA-funded research done at Yale.
Read more
A Day in the Lab with FRAXA Investigator Dr. Tue Banke
Recently Laurie Bowler and her 19-year-old son Casey, who has Fragile X syndrome, visited FRAXA research grant recipient Dr. Tue Banke at his University of Washington laboratory. We hope you enjoy Laurie’s wonderful description of their adventure! FRAXA awarded $90,000 to Dr. Banke to study the Developmental Profile of Glutamatergic Synapses in Fragile X.
Read more
Gene Therapy Translational Studies for Fragile X Syndrome
With this $90,000 award from FRAXA Research Foundation, Drs. Ernest Pedapati, Christina Gross, and student Lindsay Beasley will pursue preclinical gene therapy approaches using AAV (adeno-associated virus) vectors for treating Fragile X syndrome at Cincinnati Children’s Hospital. Dr. Craig Erickson elaborates about this in this video.
Read more
Clinical Trial of Metformin for Fragile X Syndrome
Metformin is commonly prescribed to control high blood sugar in type 2 diabetes. With a $50,000 grant from FRAXA Research Foundation, Dr. Artuela Çaku and Dr. Francois LePage are conducting an open-label clinical trial of metformin for children and adults with Fragile X syndrome, at the University of Sherbrooke in Canada.
Read more
Developmental Profile of Glutamatergic Synapses in Fragile X
FRAXA Research Foundation awarded a $90,000 research fellowship to Dr. Tue Banke. With this award, Dr. Banke investigate how glutamate receptors at neuronal synapses – essential building blocks of learning and memory – are impacted in Fragile X syndrome.
Read more
Reintroducing FMRP via Tat to Reduce Symptoms of Fragile X Syndrome
FRAXA Research Foundation and the Fragile X Research Foundation of Canada awarded a grant of $100,000 over two years to Dr. Raymond Turner at the University of Calgary in Alberta, Canada. Dr. Turner and postdoctoral fellow Xiaoqin Zhan, PhD are attempting to reactivate a segment of FMRP to reverse symptoms of Fragile X in a mouse model of the disease to reduce abnormal behaviors.
Read more
Enhancing NMDA Receptor Signaling to Treat Fragile X Syndrome
Dr. Stephanie Barnes investigated the role of NMDA receptors as a FRAXA Postdoctoral Fellow in Dr. Emily Osterweil’s laboratory at the University of Edinburgh from 2016-2018. With an additional year grant from FRAXA, she then continued her work to identify novel targets and test pharmacological therapies in the Fragile X mouse model at the Picower Institute at MIT with Dr. Mark Bear. Results published.
Read more
FRAXA Biotech Games, the Beginning of Something Great
On September 20, 2018, FRAXA Research Foundation held the First Annual FRAXA Biotech Games™. The event was a “friendly” competition between greater Boston biotech companies and affiliated industry partners and vendors in a series of fun backyard lawn games. 42 teams of 4 players each played cornhole, KanJam, ladder golf and bucketball. Our goal was to establish an annual event in Cambridge, MA, that would unite the biotech community for an afternoon of fun competition, and raise money for biomedical research. We look forward to the upcoming Biotech Games!
Read more
Federal Funding for Fragile X Research Faces Snags
We in the Fragile X community have our advocacy work cut out for us to restore levels of federal funding for Fragile X research! Both of the main federal funders – the National Institutes of Health (NIH) and the Department of Defense (DOD) are letting Fragile X research lapse!
Read more
One Person Can Make a Difference
When Ellen Skala, also referred to as Elle, contacted us in August 2018 about creating a personal fundraising campaign for FRAXA, we were excited about her interest in supporting our mission. Elle set a goal to raise $10,000 for Fragile X research by her birthday, and she committed to shaving her head if she met her fundraising goal!
Read more
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.
Read more
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.
Read more
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!
Read more
Screening 2,320 FDA-Approved Drugs for Potential Treatment of Fragile X
FRAXA Research Foundation has awarded a $90,000 grant to Principal Investigator Dr. Sean McBride and Postdoctoral Fellow Dr. Karen Joyce, at Rowan University, to screen all 2,320 FDA-approved drugs on both mouse and fly models of Fragile X syndrome. Those drugs which show promise will be tested in more detail for potential to treat Fragile X in humans.
Read more
Kathan Pierce Honored as a New England Patriots Difference Maker
FRAXA is proud to announce that one of our board members, Kathan Pierce, was recognized by the NFL’s New England Patriots and team owner Robert Kraft for her tireless volunteerism. We are grateful for Kathan’s contributions to FRAXA, and to the Patriots, who announced they are also awarding a $5,000 grant to FRAXA to support Fragile X research.
Read more
Novel Modulators of Potassium Channels to Treat Fragile X
With funding from FRAXA over 2015-2017, the Yale University team of Leonard Kaczmarek, PhD showed that the firing patterns of auditory neurons in response to repeated stimulation is severely abnormal in Fragile X mice. Based on these results, they are collaborating with the UK-based company Autifony to develop advanced compounds which may reverse these deficits.
Read more
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.
Read more
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.
Read more
Hope for the Holidays Gala Helps Advance Fragile X Research
On December 8, 2018, a sold-out crowd of 250 people gathered for the 3rd Annual Hope for the Holidays Gala at the Café Escadrille in Burlington, MA, to raise funds for Fragile X research. For the third year in a row, the Pierces presented a check for $100,000 to FRAXA Research Foundation. We are truly grateful for this ongoing, generous support of our mission to find effective treatments and ultimately a cure for Fragile X.
Read more
Coffee, Tea, and Chocolate: Adenosine Receptors in Fragile X
Caffeine is the most popular smart drug in the world. With a $90,000 grant from FRAXA Research Foundation, Alberto Martire, PhD and Antonella Borreca, PhD in Rome, Italy are investigating adenosine receptors antagonists to treat Fragile X syndrome. Compounds which are able to block adenosine receptors are commonly found in tea, chocolate, and coffee.
Read more
Boys Basketball Fundraiser Kicks Off The Season of Giving
On Saturday morning, November 17, 60 boys, their parents and a bunch of volunteers gathered in Newton, MA for the 3rd annual 3 on 3 basketball tournament, sponsored by the Newton Athletic Association (NAA), a program of Newton Community Education. The event is a fun competition of teams of three players competing for bragging rights of which team is king of the local youth basketball program.
Read more