Steve Bryson, PhD, science writer —

Neuren Pharmaceuticals has opened the first site for a U.S.-based Phase 2 clinical trial of its investigational therapy NNZ-2591 in children with Prader-Willi syndrome (PWS). “The Neuren team is very excited to be working with the community to complete this important first study of NNZ-2591 in young children…

EMO-T, a new emotional skill training program, helped children with Prader-Willi syndrome (PWS) express, recognize, understand, and regulate emotions, a study suggested. After EMO-T, scores related to emotional expression and comprehension matched those of control participants without PWS. Regulating and expressing emotions requires prerequisite emotion skills, said the researchers,…

Reduced activity of the Magel2 gene, which is defective in Prader-Willi syndrome (PWS), specifically in nerve cells that supply the brain’s amygdala, lowered the susceptibility to diet-induced weight gain by increasing physical activity. The researchers noted that these unexpected findings from a mouse study may provide a novel therapeutic strategy…

A Phase 2 study evaluating ARD-101, an investigational, gut-targeting Prader-Willi syndrome (PWS) oral therapy, is now enrolling up to 12 patients, ages 17 to 65. The open-label study, sponsored by Aardvark Therapeutics, the treatment’s developer, will be held at two sites: Children’s Hospital in Colorado, which is…

Adolescents and adults with Prader-Willi syndrome (PWS) say they struggle with constant hunger and the urge to seek food, according to an interview-based study. Participants experienced difficulties with anxiety, behavioral outbursts, changes in schedule, and schooling, with many trying to distance themselves from the syndrome. They also expressed the…

MRI brain scans of Prader-Willi syndrome (PWS) patients unexpectedly revealed nerve cells in the cerebellum of the brain activated by food cues that promote appetite, a study demonstrated. The findings suggest that the cerebellum may be a potential target for treating obesity in people with PWS. The study, “…

In a “significant subset” of  children with Prader-Willi syndrome (PWS), obstructive sleep apnea worsened after the start of growth hormone therapy, a study of patients in Australia found. “Our results offer support for the current advice to perform follow-up polysomnography [sleep breathing study] in children with Prader-Willi syndrome after…

An $11.2-million, five-year grant has been awarded by the Centers of Excellence in Genomic Science of the National Institutes of Health (NIH) to uncover high-resolution details of the entire human genome. This project should help better understand the genome’s structure and function, as well as provide important clues about the…

The hunger hormone ghrelin, when measured in infancy, may be a biomarker for predicting the early onset of scoliosis — a sideways curvature of the spine that looks like the letter C or S — in children with Prader-Willi syndrome (PWS), a study suggested. Beginning in early infancy,…

Radius Health plans to initiate a Phase 2/3 study to evaluate the safety and efficacy of its investigational therapy RAD011, a lab-made oral cannabidiol therapy to treat excessive appetite, or hyperphagia, in people with Prader-Willi syndrome (PWS). The study, called SCOUT-015, is expected to begin by the…