Marisa Wexler, MS,  senior science writer—

SPG601, an oral treatment candidate now in clinical testing, has been granted orphan drug status for fragile X syndrome in the European Union. This designation, awarded by the European Medicines Agency (EMA), is meant to incentivize companies developing treatments for rare diseases — those affecting fewer than 5 of every…

KER-0193, an oral therapy being developed as a fragile X syndrome treatment, has been awarded orphan drug and rare pediatric disease designations by the U.S. Food and Drug Administration (FDA). These designations — which follow news of positive Phase 1 trial data from a test in healthy volunteers and…

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…

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…

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…

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…

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…

KGK Science will launch a Phase 2a trial in the coming months to test NM-1001, a psilocybin microdose therapy — meaning one that contains low doses of the hallucinogenic compound — in people with fragile X syndrome. The company has received a needed exemption from the Canadian government…

Caregivers can accurately recognize signs of anxiety in people with fragile X syndrome whose ability to articulate their emotional state is limited, a new study shows. “While most people with FXS [fragile X syndrome] cannot reliably describe their internal body or emotional states to support self-reported assessment procedures, the…