News

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…

Scores of people globally are marking Fragile X Awareness Month in July with activities aimed at calling attention to the genetic condition and community needs. World Fragile X Day is on July 22. The National Fragile X Foundation is marking the month by each day sharing a fact about…

Allos Pharma has met with the U.S. Food and Drug Administration (FDA) to optimize the design of a planned Phase 3 trial to test its investigational oral therapy arbaclofen in individuals with fragile X syndrome. The trial is intended to confirm promising findings from a previous Phase 3 study…

Attention-deficit/hyperactivity disorder (ADHD) is more common among female carriers of a fragile X premutation who have more cell-to-cell variation in the number of mutational CCG repeats in the premutation, a new study reveals. Indeed, according to researchers, “the degree of [genetic] instability (expansion) was significantly higher … in participants…

Long-term treatment with Zygel, Zynerba Pharmaceuticals’ experimental cannabidiol (CBD) gel, is tolerated well and leads to clinically meaningful behavioral improvements in children and adolescents with fragile X syndrome. That’s according to an interim analysis of nearly four years of data from the open-label extension (OLE) study ZYN2-CL-017…