News

With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails millions of Americans whose rare diseases have already been diagnosed. That’s the warning from Christopher P. Austin, MD, director of the National Center for Advancing Translational Studies (NCATS) at…

Zygel (ZYN002 CBD gel) has been granted fast track designation by the United States’ Food and Drug Administration (FDA) for the treatment of behavioral symptoms in patients with fragile X syndrome, its developer Zynerba Pharmaceuticals announced. A fast track designation is granted to a newly developed…

An Indiana University-affiliated venture fund has invested $200,000 in Confluence Pharmaceuticals to continue development of the company’s lead product for the treatment of fragile X syndrome. Along with AOP Orphan Pharmaceuticals, Confluence is developing a novel, clinically discovered adult and pediatric therapy for social and communication impairments…

A computer-based cognitive training program called Cogmed can improve working memory and cognition and increase the attention span of children and adolescents with fragile X syndrome, according to a clinical trial. Findings from the trial were published in the study, “Cognitive training for children and adolescents with fragile…

A newly developed version of the gene editing technology CRISPR — a non-viral delivery system called CRISPR-Gold — can effectively edit an autism-associated gene in a mouse model of fragile X, reducing the exaggerated repetitive behaviors common with the disorder. The study, “Nanoparticle delivery of CRISPR…

A mindfulness program run on a free mobile app may be a feasible way of easing stress in mothers of children with fragile X syndrome (FXS), especially the many who carry the disease-causing mutation passed on to their child, a study reports.

Implementing behavioral treatment via telehealth is a feasible and potentially cost-effective strategy for treating behavioral problems in boys with fragile X syndrome, a study shows. This strategy is able to circumvent the problem parents face when living in a rural area and cannot  drive long distances to reach the nearest…

Caregivers of younger children with fragile X syndrome (FXS), and those with lower family incomes, report more difficulties in accessing specialized healthcare in the U.S., a recent study found. Moreover, nearly 40% of the caregivers surveyed think their child’s doctor is…

Treatment with a compound found in green tea leaves significantly improved cognitive function in patients with fragile X syndrome, a study shows. This new clinical data came from a study of the epigallocatechin-3-gallate (EGCG) compound, a major component of green tea leaves. The study, “A phase 1, randomized…

Increased social avoidance during infancy and early childhood in boys with fragile X syndrome (FXS) is associated with higher risk of autism spectrum disorder (ASD) and lower risk of attention deficit/hyperactivity disorder (ADHD) and anxiety, according to a recent study. The study, “Infant Social Avoidance Predicts Autism but…