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The U.S. Food and Drug Administration (FDA) has granted fast track designation to Spinogenix‘s experimental oral therapy SPG601 to treat fragile X syndrome (FXS). The designation aims to speed the development and review of new medicines that have the potential to improve medical care for serious conditions. It…
Researchers in Kansas and California will investigate people with the FMR1 premutation who are at risk of developing fragile X-associated tremor/ataxia syndrome (FXTAS), a condition related to fragile X syndrome that develops late in life. The five-year project, supported by a $3.1-million grant from the National Institutes of Health, will…
A Phase 2 clinical trial to test MRM-3379, an experimental oral treatment for fragile X syndrome, is expected to launch in 2025. Mirum Pharmaceuticals announced the plan in a company press release reporting financial results and business updates. It didn’t provide details on the trial’s design…
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…
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