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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…
School-age girls with fragile X syndrome show significantly poorer cognitive function and academic performance relative to age- and IQ-matched girls without the disease, a new U.S. study reports. The fragile X group showed impairments in several cognitive skills, including nonverbal reasoning, visual pattern recognition, attention, memory, and math skills.
Signs of hyperarousal, or overreactions to sensory stimuli, and abnormal processing of sensory information — including difficulty with eye gaze and sensitivity to touch and certain sounds — are highly prevalent among people with fragile X syndrome, a new study reports. Such overreactions and resulting behavioral difficulties were found…
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease (RPD) designation to zatolmilast, Tetra Therapeutics’ investigational oral therapy for fragile X syndrome. The designation is given to experimental therapies with the potential to provide clinically meaningful benefits to patients, primarily those younger than age 18,…
Screening of potential participants has begun in a Phase 2a trial that will test Nova Mentis Life Science’s NM-1001, a microdose of the psychedelic molecule psilocybin, in adults with fragile X syndrome. The study (NCT05832255), which is being conducted in a partnership with KGK Science,…
PureTech Health is planning a Phase 2 clinical trial to test its lead therapeutic candidate LYT-300 — an oral formulation of allopregnanolone — in people with fragile X-associated tremor/ataxia syndrome (FXTAS). The company has been awarded a grant of up to $11.4 million from the U.S. Department of Defense…
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