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With $1.6 million in pre-seed funding, researchers have launched a new biotech company called Aptadir Therapeutics that will develop RNA-based therapies to reactivate specific genes that are silenced in cancer and genetic diseases such as fragile X syndrome. The therapies will be based on a new class of RNA…
The nonprofit FRAXA Research Foundation has teamed up with Marvel Biosciences to test MB204, a drug that blocks adenosine A2A receptors, in a preclinical model of fragile X syndrome. As part of the collaboration, FRAXA will run the preclinical studies at its…
Tetra Therapeutics has amended the protocols of two ongoing late-stage clinical trials testing zatolmilast, its experimental oral therapy for fragile X syndrome, to improve access for patients and their families. The similarly designed studies are recruiting up to 300 male fragile X patients at 15 sites across the…
July is Fragile X Awareness Month, and this year, there are many ways to help build and spread knowledge about fragile X syndrome, collect funds for research, and advocate for those living with the rare genetic disease. To encourage people to stay engaged in the countdown to World Fragile…
Patients, caregivers, and professionals will gather next month at the National Fragile X Foundation‘s (NFXF) 19th International Fragile X Conference to learn about the latest updates in fragile X syndrome care and research and help empower those living with the rare disease. The conference will be held…
The U.S. Food and Drug Administration (FDA) has given Spinogenix the green light for a Phase 2 clinical trial that will test its investigational oral therapy SPG601 in adult men with fragile X syndrome. “Current treatments leave a critical gap in effective and patient-friendly solutions for neurodevelopmental conditions,”…
The LivJoy Foundation is funding a four-year postdoctoral research fellowship for an early career scientist at the University of South Carolina (USC) who will study fragile X syndrome in females. The sponsorship was borne from a shared vision between Rachel and Brian Clouse, who started the Foundation in…
A combination of therapeutic candidates gaboxadol and ibudilast normalized behavior and improved memory in a mouse model of fragile X syndrome in a new study. The study, “A novel combination treatment for fragile X syndrome predicted using computational methods,” was published in Brain Communications. The work was…
Researchers in Egypt have uncovered two biomarkers that could help identify fragile X syndrome where genetic testing is not possible. Among patients with suspected fragile X based on symptoms, blood levels of two proteins — metalloproteinase-9 (MMP9) and amyloid-beta protein precursor (APP) — were higher in those where…
Excessive DNA repeats in the FMR1 gene — the cause of fragile X syndrome — lead to structural changes in other parts of the DNA that disrupt the activity of genes necessary for nerve cell function, according to a study using samples from fragile X patients. This damaging effect…
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