Meet Archie!

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

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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Potential Upcoming Advances in Fragile X Research

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

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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Reactivating the Fragile X Gene in Young Mice Reverses Symptoms

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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10 Year Vision for Collaborations That Transform Fragile X and Autism Research

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 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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Characterization of a Novel CYFIP1 – Derived Peptidomimetic Restoring the Dysregulated mRNAs Translation: Toward An Innovative Therapeutic Strategy for FXS

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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FX-Learn Clinical Trial for Children with Fragile X

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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Cannabinoids as a Treatment for Fragile X Syndrome

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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New Fragile X Clinical Trial Announced by Healx

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 successful 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.

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Correcting the Brain’s Emotional Memory Center

Correcting the Brain’s Emotional Memory Center

ell 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

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 upcoming trial, which will 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

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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Meet Matthew!

Meet Matthew!

Meet #FriendofFRAXA Matthew! He is in his final year of school and has always been enthusiastic about his education. He has faced each day with a positive attitude while being friendly and outgoing. Those who meet Matt say he leaves a lasting impression on them. He has been called “The Mayor”, as everyone knows and remembers Matthew where ever goes.

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7th Annual 5K Walk for Fragile X in NH Raises Over $3200 For Fragile X Research

7th Annual 5K Walk for Fragile X in NH Raises Over $3200 For Fragile X Research

Derry, New Hampshire served as the picturesque location for the 7th Annual 5K Walk for Fragile X syndrome and Raffles to support FRAXA Research Foundation. On September 12, 2021, around 50 individuals came together to show their support and make a difference in the lives of those with Fragile X syndrome. This year’s walk, which included raffle drawings with baskets and gift cards thanks to the generosity of local businesses and friends, raised over $3,200 for FRAXA to support the funding of effective treatments and ultimately a cure for Fragile X.

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