FRAXA Research Foundation

FRAXA is pleased to announce a $100,000 research grant to Beverly Davidson, PhD and Carolyn Yrigollen, PhD at Children's Hospital of Philadelphia. The team aims to optimize state-of-the-art gene editing tools to treat Fragile X syndrome. They will test these tools in a unique mouse model to see if they can correct FMR1 mutations. This research will advance our understanding of how to use gene editing to treat Fragile X syndrome, and it’s made possible by the generosity of our supporters. Thank you for helping us drive these advances forward! Dr. Beverly Davidson is Director of the Perelman Center for Cellular and Molecular Therapeutics and Chief Scientific Strategy Officer at Children’s Hospital of Philadelphia. Dr. Carolyn Yrigollen is a Research Associated Scientist II at Children's Hospital of Philadelphia (CHOP) and a FRAXA Postdoctoral Fellow in Dr. Davidson's lab. #FragileX #FXResearch #GeneEditing #NeurologicalDisorders #ScientificResearch #GeneTherapy #InnovationInScience #Neuroscience #ChildrensHospital #AdvancesInMedicine

FRAXA is proud to announce a $100,000 research grant awarded to Dr. Craig Erickson, Dr. Lisa De Stefano, and Dr. Lauren Schmitt at Cincinnati Children's Hospital. This grant aims to transform clinical trials in Fragile X syndrome by simplifying and adapting EEG technology for home use. By enabling clinicians to administer EEG tests in patients' homes, this project could significantly improve the efficiency and accessibility of future trials. Thank you to our generous donors who make these crucial advances in Fragile X research possible. #FragileX #FXResearch #ClinicalTrials #Neuroscience #EEG #BrainHealth #RareDiseaseResearch #InnovationInHealthcare #CincinnatiChildrens #HomeBasedCare

FRAXA Research Foundation is working together with Marvel Biosciences Corp. in an exciting new preclinical study examining the effects of Marvel’s MB204 drug. FRAXA’s collaboration with Marvel Biosciences exemplifies FRAXA's strategy of feeding new treatment ideas into the development pipeline. By funding high-quality academic studies with a therapeutic focus and partnering with innovative companies like Marvel, FRAXA accelerates the development of promising new treatments for Fragile X syndrome. #FragileX #FragileXResearch #FragileXTreatment #DrugDevelopment #ScientificResearch #Innovation

We're happy to share this exciting announcement from Marvel Biosciences Corp. Marvel will test their experimental new drug at FRAXA's Drug Validation Initiative (FRAXA-DVI) to see if it has potential to treat Fragile X syndrome. This partnership builds on FRAXA-funded research conducted in Italy that explored adenosine receptor antagonists for Fragile X. #FragileX #FXResearch #DrugDevelopment #PharmaCollaboration #InnovativeScience #AdenosineReceptors #Neuroscience #ClinicalResearch #RareDiseaseResearch #BiomedicalResearch

🌍 On July 22, 2024, World Fragile X Day brought together 13 countries in a remarkable display of global unity and awareness. From Niagara Falls to Matagarup Bridge, over 400 landmarks around the world were illuminated to symbolize hope and solidarity for those affected by Fragile X syndrome. As we look back on this impactful day, we're reminded of the power of global collaboration in raising awareness and supporting the Fragile X community. Let's continue to shine a light on Fragile X syndrome and work together towards a brighter future. #FragileX #WorldFragileXDay #GlobalAwareness #CommunitySupport

Discover invaluable insights on managing anxiety in individuals with Fragile X syndrome from our recent World Fragile X Day webinar. Dr. Carrie Buchanan, Fragile X Program Director at Greenwood Genetic Center, delves into the neurobiological reasons behind anxiety, common anxiety behaviors, and practical strategies to enhance the quality of life for children and adults with Fragile X. Understand the role of the amygdala, non-medication treatments, and medication options in this informative session. #FragileX #FXResearch #FragileXAwareness #AnxietyManagement #Neurobiology #ChildHealth #GeneticResearch #MentalHealthSupport #TherapeuticInterventions #ClinicalTrials

We are excited to announce that a $100,000 FRAXA research grant has been awarded to Xinyu Zhao, PhD, and Zhiyan Xu at the University of Wisconsin-Madison. This grant will support their study, "To Interrogate the Developmental Timing for Treating Fragile X Syndrome." Are there specific ages when treatment is most effective? Can treatments work as well for adults as they do for children? With this grant, the team will conduct studies to answer these important questions. Thank you to all our donors for making this important research possible! #FragileX #FXResearch #FragileXsyndrome #ResearchGrant #ScienceForGood #GeneTherapy #DrugTreatments #DevelopmentalTiming #Neuroscience #MitochondrialResearch

We are delighted to share the success of the 37th annual charity golf tournament organized by the Hall family. This year, they raised over $35,000 for Fragile X research! Their ongoing efforts have contributed over $327,000 to FRAXA, driving forward research advances. Thank you to all the sponsors, participants, and volunteers for your incredible support. Together, we are making significant strides toward finding a cure for Fragile X syndrome. #FragileX #FXResearch #CharityGolf #ResearchAdvances #CommunitySupport #GolfForACause #FundraisingSuccess #ScientificBreakthroughs #NonprofitEvents #ThankYou

Kaerus Bioscience Ltd. has initiated a Phase 1 clinical trial for KER-0193, a novel BK channel modulator developed for Fragile X syndrome, in Belgium. After over 5 years of R&D, supported by partnerships with academic collaborators and FRAXA, this trial marks a significant milestone. KER-0193 has shown promising preclinical results in improving behavioral, sensory, and cognitive functions. FRAXA is proud to have been part of this journey and we look forward to seeing the outcomes of this clinical trial. #FragileX #FXResearch #ClinicalTrial #BKChannel #behavior #sensory

Today Shionogi announced significant progress in their efforts to develop the investigational new drug Zatolmilast (BPN14770) to treat Fragile X syndrome (FXS). Zatolmilast has received Fast Track, Rare Pediatric Disease, and Orphan Drug Designations from the FDA, as well as Orphan Medicinal Product designation from the European Commission. Large scale pivotal clinical trials for individuals with Fragile X are ongoing across the U.S. Key changes to these trials: -- Lowered minimum age for trial participants -- Integration of remote visits to reduce on-site requirements -- Extended open-label extension period to up to two years -- Coverage of travel expenses for participants and caregivers   We are grateful to all the families participating in clinical trials! Your involvement is crucial in driving progress and bringing new treatment options to our loved ones and future generations. FRAXA supported the early development of zatolmilast with multiple research and clinical grants. #FragileX #ClinicalTrials #FXResearch #Zatolmilast #WFXD #HopeForFXS #PatientAdvocacy #InnovativeMedicine https://lnkd.in/emGaa8tB