Lindsey Shapiro, PhD, science writer —

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…

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…

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,…

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…

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…

Cell growth and signaling, as well as cholesterol production, are significantly altered in lab-grown astrocytes — the star-shaped brain cells that provide support to neurons — derived from people with fragile X syndrome, a study shows. The patient-derived cells also had altered levels of a number of proteins that…

KGK Science is requesting permission from Health Canada to conduct a Phase 2a clinical trial evaluating a microdose of the psychedelic compound psilocybin in people with fragile X syndrome. This first in-human trial seeks to investigate the safety of this psilocybin microdose, called NM-1001, as well as its…

A month of treatment with retinoic acid — a naturally occurring byproduct of vitamin A — was able to restore certain social behaviors in a mouse model of fragile X syndrome, according to a recent study. The molecule likely restored the behaviors by normalizing levels of certain genes in…