Latest Fragile X Research Grants & Insights from Recent Scientific Meeting | May 2024 Webinar

Описание: Join us for an in-depth look at new developments in Fragile X research from our FRAXA Research Q&A Webinar held on May 23, 2024. This recording, featuring Dr. Michael Tranfaglia, MD, Medical Director and Co-Founder of FRAXA, provides valuable updates on current Fragile X research.

In this session, Dr. Tranfaglia shares insights from the latest Fragile X scientific meeting, and we discuss the newest batch of research grants awarded by FRAXA. While this video focuses on research updates, a separate video will cover current clinical trials.

Whether you're a researcher, healthcare professional, or part of a family affected by Fragile X, this video offers a wealth of information and the opportunity to hear from leaders in the field.

Highlights
-- Insights from the latest Fragile X scientific meeting
-- Announcement of new Fragile X research grants
-- Q&A with Dr. Michael Tranfaglia and Katie Clapp

Don't miss out on this chance to learn about the progress being made in understanding and treating Fragile X syndrome. Watch now and stay connected with FRAXA Research Foundation for more updates.

Subscribe to our channel for more videos on Fragile X research and clinical trials, and visit our website at FRAXA.org for more resources and information.

Chapters
0:00:00 *Insights from the Latest Fragile X Scientific Meeting*
0:03:55 *2024 FRAXA Research Grants Awarded*
0:04:40 R-loop-mediated CGG Contractions as a Potential Therapeutic Approach
0:10:35 Gene Editing of FMR1 to Correct Fragile X Syndrome Phenotypes in Mice
0:12:50 Identification of the Proteome of Active and Silence FMR1 Alleles in Human Stem Cells using an Unbiased Proximity Biotinylation Screen
0:14:21 Small-Molecule Inhibitors of Nonsense-Mediated mRNA Decay (NMD) to Treat Fragile X Syndrome
0:16:12 Investigating and Correcting the Mechanistic Basis of Altered Physiology of Primary Visual Cortex in Fragile X Syndrome
0:18:08 Role of the Insulin Degrading Enzyme (IDE) in Fragile X Syndrome
0:19:27 To Interrogate the Developmental Timing for Treating Fragile X Syndrome
0:22:11 Probiotic Intervention in Children with Fragile X Syndrome: Pilot Study
0:24:13 Fragile X Unplugged: Establishing Mobile EEG as the Next Frontier
0:27:20 *Question & Answers*
0:27:30 What is the status of research into ASOs?
0:32:53 What is the best way to learn and track what Fragile X researchers are doing?
0:34:13 What is the current status of CRISPR Cas9 and when can we expect the first clinical trial with it?
0:37:12 Do we have a credible combination of drugs that will reduce the symptoms of Fragile X?
0:44:53 Can AI help in finding treatments for Fragile X syndrome?
0:49:10 If Fragile X is the cause of autism in a person and Fragile X is cured, is autism then cured?
0:49:55 How much will treatments or a cure help mid-life adults with Fragile X?
0:55:12 Which Fragile X research group is closest to having a cure come to market?
0:59:39 Wrap-up

FRAXA’s mission is to find effective treatments and ultimately a cure for Fragile X syndrome. We directly fund research grants and fellowships at top universities around the world. We partner with biomedical and pharmaceutical companies, large and small, to bridge the gap between research discoveries and actual treatments.

Treatments for Fragile X are likely to help people affected by autism, Alzheimer’s, and other brain disorders.

To learn more about FRAXA's Fragile X syndrome research, to get involved with Fragile X awareness and events or to donate to FRAXA research, visit www.fraxa.org

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