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.
Thank you for sharing. We're here to support you and the entire Fragile X community. If you're looking for resources or information, feel free to explore our website (www.fraxa.org) or reach out.
Thank you. I had a child in my home for a couple of years that we tested for this...he didn't have it, but he had the ears and the developmental slowness. I passed out of his life, and wonder how he came out---we were seeking answers then.
Thank you for sharing that experience. It sounds like you were deeply involved in his care and search for answers, which makes such a difference. It's understandable to wonder how things turned out for him. Fragile X can be a complex journey, and there are many factors at play in developmental challenges.
Nova Mentis Life Sciences (NMLSF) started on some kind of clinical trials for curing Fragile X over a year ago, but I have heard nothing since their "BIG" announcement. Does anybody have an update on their results?
I'm getting into Fragile X research as a PhD student! Looking forward to following this channel and hopefully contributing to this research in the future
That’s exciting! Welcome to the Fragile X research community! We’re thrilled to have you following our work and look forward to seeing your contributions in the future. Best of luck with your PhD journey-together, we can advance Fragile X research!
Does insurance cover that genetic testing Fragile X? I was diagnosed w/Ehlers Danlos but I was never tested for the marker- it was clinical as I had all of the symptoms of Ehlers Danlos Syndrome. I’ve also been diagnosed with ADHD as a teen & Nonverbal Learning Disorder . Mild Ataxia, low muscle tone as well as Strabismus. From ages 18 months I had to wear orthotics & orthopedic shoes (hated those shoes) & had wear bifocals sun I was a toddler. Anxiety , depression, sensory processing disorder. What I understood is that Fragile X is the most common cause of Autism in the Ashkenazi community/ethnicity but not so much in the general population. So, I’m a bit confused Since I’m over the age 23, I don’t get any support. So, is there any benefit for me to get evaluated?
Thank you for your question! Insurance coverage for Fragile X genetic testing varies, so it’s best to check with your provider. A genetic diagnosis, even as an adult, can offer valuable insights into managing symptoms like anxiety, ataxia, and sensory processing disorder. It may also help your healthcare team explore new treatment options. Consulting with a genetic counselor or your doctor could be a good next step. Here is some more info about testing for Fragile X syndrome: www.fraxa.org/fragile-x-syndrome/cause/#testing-diagnosis
My step brother has this. His intellectual age is between 9 and 12, depending on the skill. He can add, but not subtract, he reads at a first grade level, and doesn’t have the skills to live without social service help. However, he does know that he is an adult, even though he may not know his age. Thank you for this video!
Thank you for reaching out with your question. The investigational new drugs we're discussing are specifically being tested for treating Fragile X syndrome. While they offer hope in this area, they are not currently anticipated to be particularly effective for individuals with the Fragile X premutation.
They are currently recruiting for their large scale phase 3 clinical trial. www.fraxa.org/tetra-therapeutics-phase-3-clinical-trial-of-zatolmilast-in-fragile-x-syndrome/
@@scentedgamer8676 phase 3 started recruiting late 2022. Typical phase 3 studies can last 1-4 years. It really depends on how quickly they can get all of the study participants enrolled.
The recent webinar linked below has more details about the this trial and the other late stage trial currently going on. ru-vid.com/video/%D0%B2%D0%B8%D0%B4%D0%B5%D0%BE-hkAcrDD0Lyk.htmlsi=WvUiy5aDxwZgWCI3
Phase 3 clinical trials typically last between one to three years. This phase involves a larger group of participants and focuses on confirming the effectiveness of the treatment, monitoring side effects, and comparing it to standard or placebo treatments. The exact duration can vary based on the specific objectives, the number of participants, and the endpoints being studied.
My nephew has fragile x...his father is on the streets refusing help off drugs and his mother died of cancer..I just got a message from cps today after years no contact saying he needs a place or he's going to foster care😢😢😢😢😢
dont lose hope, the stem cells therapy is available in countries like India, china, mexico.. i was treating my daughters autism, i didnt know she had fragile x.. she is improving a lot.. here they r still researching.. i dont understand y they r not collaborating studies results.. they r still studying gut microbiome.. in india they r already doing it! i wish India focussed on fragile X
@scentedgamer8676, Sorry to hear you feel this way. Research progress can be slow, but every step brings us closer to treatments and a cure. FRAXA remains committed to advancing research and supporting the community. The dedication and persistence of researchers, advocates, and families are driving forces behind our efforts. Together, we can stay hopeful and keep pushing forward.
@@FRAXA i know its slow and understand why.I also know that fraxa does their best.I have been following fraxa for over 10 years since mark bears solution failed and zynerba as well,but when i saw this video about the dosages vary from person to person there will never be a fda aproved treatment for now,maybe in 100 years or something but not in the near future.
Hi, Thanks for asking! We just uploaded half of it. We'll have the 2nd part up later this week. ru-vid.com/video/%D0%B2%D0%B8%D0%B4%D0%B5%D0%BE-7PLWjRkPiPA.htmlsi=ji6hiulBz9czHlhx
The trial is still progressing. Zynerba has been acquired by Harmony Biosciences and are pushing the trial forward without any interruptions. fragilexhelp.com/
We are moving forward and excited about the future. We remain focused on our goal of effective treatments and ultimately a cure. There are many options in the pipeline and we continue to explore all avenues that show promise. The more runners in the race the better odds one finishes.
Starting to think that I have this fragile X syndrome.. Don't think this existed back in the '80s when I was born and diagnosed with ADHD but I also have a lot of social issues I don't like being in large groups with a lot of people, I am generally happy and laughing, along with other anxiety issues I'm 41 yrs old now when I was young in school I had behavioral issues and learning issues, They put me in the bad kid classes instead of the learning disability ones because My intellect was between regular kids and kids with learning disabilities
as an autistic: trust me, neurotypicals (aka the "average ppl") are actually the weak ones with disabilies like low IQ, lack of empathy and other symptoms. neurodivergent folks are geniusses. don't trust researchers and better check out what the system does to us (there are many names, for example hans asperger, just check it out where this whole topic is rooted. humanity is sick...) I bet you'll understand as soon as you see the pattern. also check out temple gradin, she is brilliant, her mind full of facts. don't trust most ppl, their brains are TOTALLY different wired as well as the rest of our body. they won't and can't understand (because not smart + being manipulated by the psychopathic people of the system)
Thank you very much for the inside, Mr Larson 🙂 I am studying for an pediatric exam (becoming an OT) right now and those little facts really help me to memorize 😊 I wish you all the best, Philipp
We understand how difficult this journey can be, and your son’s story motivates us every day to push forward with research. Thank you for your kind words, and we’re here for you and your family.
This is concerning. Is there a special genetic test you have to get besides a normal baby screening at birth or a genetic test like at 23andMe? I'd like to know if my little one, who is 14 months old, could have it. Health issues that have been mentioned run in my family, but of course a lot of other things could cause these symptoms. Bring a mommy now just worries me since my baby was born 2 months early because he had to come out or we both could have died. I'm not sure if things I've noticed are other health issues or something like this mutation. Thank you for posting this and letting others know.
Hi, There are testing options: www.fraxa.org/fragile-x-syndrome/cause/#testing-diagnosis Please use a medical geneticist at a local as medical facility as they will be able to help you best.
Check out our recent webinar were we give some updates and progess on a CBD clinical trial. ru-vid.com/video/%D0%B2%D0%B8%D0%B4%D0%B5%D0%BE-hkAcrDD0Lyk.htmlsi=7asneE8DvVf6RW6M
I cannot tell you how much I relate to these symptoms, I don't have an intellectual disability, but I have been playing whack a mole with something my whole life. Learning disabilities, adhd, Visual processing disorder, and my horrible writing quality And so much more, I cannot find an underlying cause, I would love to be tested for this but I am not convinced that going to my dr and saying I have done 10 minutes of internet research and this is what I have is a good idea.
Hello, we’re in Toronto, Canada. Our 15 year old son has full mutation FXS with what we think is mosaic. We’re interested in Tetre trials. How can we participate from Toronto?
Hello. You would need to be able to travel to one of the US clinics for visits. Travel by air might be covered by the trial sponsor. If that travel might be possible for you, please send us an email at info@fraxa.org and we can get you more details. The clinics are listed at www.fraxa.org/tetra-therapeutics-initiates-phase-3-clinical-trial-of-zatolmilast-in-fragile-x-syndrome/
We still have much to learn, but have come so far in understanding fragile X and other genetic conditions. It's truly amazing the advances happening currently. It's very exciting and hopeful. Thank you!
Have a good time. I have an 8-year-old child who has Fragl X. I follow you cordially. Perhaps you will discover a cure for this disease and that my child will become normal. In previous years, you were told that you have found that a protein is responsible for this imbalance. Unfortunately, the effect of this injection is for one day only, and you will develop this protein. Any news ? Unfortunately, all episodes are about donations!!! And the most important question is, do routine vaccinations for children, especially MMR, cause these diseases, including autism????
That was an excellent explanation of the genetic disorder. Just one question. You mentioned that the mutation causes a failure to produce a particular protein. Is it possible to synthesize the protein and supply it to the patient to offset the deficiency or is it more complicated than that? You mentioned the research going into trying to cure the condition via genetic engineering methods, but you didn't comment on whether the protein can be synthesized.
