The hardest part about making the mittens is finding wool sweaters. It takes Carol 2 – 2 ½ hours to complete a pair of wool mittens, which have a soft fleece lining. Mitten sales have done very well over the years, enabling Carol to support Fragile X research.
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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.
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Correcting Fragile X Syndrome Deficits by Targeting Neonatal PKCε Signaling in the Brain
With this $90,000 grant from 2017-2018, Dr. Banerjee’s team has shown that enhancing PKCε can correct brain development and abnormal behaviors in Fragile X knockout mice and had their findings published in PubMed.
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Climbing 3 Mountains for Fragile X Research
People often say they would do anything for their children. What if that “anything” was to climb a mountain? How about three in a twenty-four hour time period? That’s exactly what Michael Leonard did in honor of his son, Cole, who lives with Fragile X syndrome.
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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.
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Lovamix: Clinical Trial of Combined Treatment of Minocycline and Lovastatin in Fragile X Syndrome
With a $66,714 grant from the FRAXA Research Foundation awarded over 2015-2017, Dr. Francois Corbin at the Universite of Sherbrooke will test the safety and synergistic effects of lovastatin and minocycline in patients with Fragile X syndrome.
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FRAXA’s Most Successful End-Of-Year Campaign Ever!
FRAXA Research Foundation relies on the generosity of others to make our shared dream a reality. Our 2021 annual appeal was FRAXA’s most successful end-of-year campaign EVER! Together we will find effective treatments and ultimately a cure for Fragile X syndrome.
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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.
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Reactivating the Fragile X Gene in Young Mice Reverses Symptoms
A new FRAXA-funded research project offers hope that Fragile X syndrome could be treated by reactivating the gene which is shut down in people with the syndrome. Researchers at the University of California, Riverside report that they were able to reduce FXS symptoms by inserting the FMR1 gene into the brains of very young mice.
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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.
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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.
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Patrick’s PALS Still Shined Strong Even in the Face of COVID
Patrick’s PALS 25th Annual 3-on-3 Basketball Tournament & Fundraiser was once again relegated to “Fundraiser Only” status in 2021. BUT, and it’s a BIG but, the spirit, generosity and devotion of so many to this event still shined through. Over $140,000 was raised through a mail campaign, all of it of course going to FRAXA to continue to advance research that will someday lead to effective treatments and ultimately a cure.
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16th Annual Fragile X Poker Run Raises nearly $29,000
The 16th Annual Fragile X Poker Run took place July 31 – August 2, 2021 in North Carolina. The generosity of the participants raised nearly $29,000 for FRAXA and Fragile X Research!
Great family and friends from 10 different states attended the event, one friend even came from Israel!
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GABA-A Receptor in Fragile X Syndrome
With $210,000 in grants from FRAXA over 1999-2010, Dr. Frank Kooy has studied the involvement of the GABAergic system in Fragile X syndrome, at the University of Antwerp, Belgium.
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Characterization of a Novel CYFIP1 – Derived Peptidomimetic Restoring the Dysregulated mRNAs Translation: Toward An Innovative Therapeutic Strategy for FXS
The researchers are developing next-generation drugs called peptidomimetics, using the functional features of FMRP. If they succeed, the hope is that we will have new drugs that could make up for the loss of FMRP, thus treating Fragile X syndrome.
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Bruins 50/50 Benefits Fragile X Research
As the Bruins took to the ice to score a big win over the Detroit Red Wings, FRAXA volunteers took to the concourse selling 50/50 tickets to fans. This incredible opportunity, made possible by the Boston Bruins Foundation, raised a grand total of $28,040 which was split between FRAXA and one lucky winner!
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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.
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Over $20,000 Raised For Fragile X Research at Callum Cup V
After a one year hiatus, due the COVID-19 pandemic, the Callum Cup made a triumphant return last month. This annual intra-club charity match is the marquee event of the Millburn FC soccer calendar. The game being played in honor of Callum Murphy, the son of Andrew Murphy, one of the club’s goalkeepers.
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Cannabinoids as a Treatment for Fragile X Syndrome
Many people with Fragile X syndrome are hyper-sensitive to sights and sounds, and Electroencephalography (EEG) studies show that there are abnormalities in brain circuits. EEG studies show similar changes in Fragile X mice. So the team will use EEG tests in mice to find which drugs best reduce hypersensitivity. They can then easily move on to human EEG-based clinical trials. What they learn will tell us much more about why people with Fragile X are hypersensitive – and which drugs could best help them.
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Fundraising Never Tasted So Good
I scream, you scream, we all scream for ice cream! That’s what over 100 people said on Sunday, September 26 when the Lancaster Sweet Shoppe in Lancaster, Pennsylvania donated all of its proceeds from the day to FRAXA Research Foundation, in honor of my son, JT. All said and done, we are investing $15,205.30 in FRAXA’s work.
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New Fragile X Clinical Trial Announced by Healx
Healx’s AI-driven approach makes finding the right combination therapies more efficient, cost-effective, and rapidly ready for testing at FRAXA-DVI. It was this process that has brought Healx to its recent announcement sharing that it has received Investigational New Drug (IND) approval from the US Food and Drug Administration (FDA) for the Phase 2a clinical study of HLX-0201 (sulindac, an FDA-approved drug).
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Forbes, “Clinical Trials Expand For AI-Designed Drug To Treat Fragile X Syndrome”
British startup Healx has secured FDA approval for a phase 2a clinical trial of an AI-discovered compound that could help manage the symptoms of the genetic disorder Fragile X syndrome. The start of the trial marks another milestone in the use of artificial intelligence to help find new applications for existing drugs by mining patient records and research databases.
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Correcting the Brain’s Emotional Memory Center
Cell Reports has published the results of the study “Correction of Amygdalar Dysfunction in Rat Model of Fragile X Syndrome” by FRAXA Investigator Dr. Sumatra Chattarji and his team at the National Centre for Biological Sciences in Bangalore, India. The researchers investigated the synaptic basis of deficient conditioned fear and its reversal in Fragile X syndrome rats.
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Purposeful and FRAXA Partnership Leads to Clinical Trial
Can a combination of drugs make a meaningful difference for people with Fragile X? A new clinical trial is going to find out. 15-20 adult men with Fragile X will be included in this trial to test the effects of an available drug and a nutritional supplement taken together.
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Making Drug Development Efficient Through Community-Based Collaboration
FRAXA co-founders Katie Clapp and Mike Tranfaglia, spoke virtually at the 5th Pharma Pricing Reimbursement and Market Access 2021 conference.
In this session, facilitated by Nadia Bodkin, PharmD, MS, from the Rare Advocacy Movement, Katie and Mike were joined by Christopher U. Missling, PhD, President and CEO of Anavex Life Sciences Corp. to discuss the collaboration between FRAXA and Anavex as a case study example to help raise awareness amongst others in the rare disease industry of these types of collaborations between advocacy and industry.
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