News

Neuren Pharmaceuticals will test its therapeutic candidate NNZ-2591 in children with Prader-Willi syndrome (PWS) in a new Phase 2 clinical trial, the company announced in a press release. The U.S. Food and Drug Administration (FDA) gave Neuren the go-ahead to conduct the trial following the company’s…

Children with Prader-Willi syndrome (PWS) have less lean body mass and grip strength, and spend more time inactive compared to healthy children or those with non-alcoholic fatty liver disease, a pilot study in Canada has found. “These findings are relevant as early detection of deficits in muscle strength and…

A boy was found to manifest “blended” symptoms of both Prader-Willi syndrome (PWS) and a form of hereditary spastic paraplegia caused by a double copy of a faulty chromosome he inherited from his mother, according to a report from the U.S. The report, “Blended Phenotype of Prader-Willi Syndrome…

The Foundation for Prader-Willi Research has awarded a two-year, $141,444 grant to help fund the development of a patient-reported questionnaire to assess the impact of hunger for people with Prader-Willi syndrome (PWS). The project is being led by Elisabeth Dykens, PhD, a professor of psychology…

Compared to their typically developing peers, children with Prader-Willi syndrome show marked differences in the shapes and sizes of many structures in the brain, including some important for regulating hunger, a new study reports. Changes seen in patients were not seen in obese children who didn’t have Prader-Willi, indicating…

Researchers have determined that the role of the chemical messenger oxytocin in Prader-Willi syndrome could be associated with its function in astrocytes, a type of nerve support cell in the brain. By counting the number of oxytocin-producing cells and receptors for the molecule in the brain, brain regions and cell…

Treatment with liraglutide along with an exercise and diet program eased excessive hunger in children and adolescents with Prader-Willi syndrome (PWS), but had no effects on body mass index (BMI) and other weight-related measures, according to the findings of a Phase 3 trial. The study, “Liraglutide…

A toddler with both Prader-Willi syndrome (PWS) and Moebius syndrome was successfully treated for respiratory distress with a cystic fibrosis medication called dornase alfa and hypertonic saline delivered via the nose. The girl displayed a rare combination of the two congenital diseases — meaning they were present at birth…

A one-time delivery of the brain-derived neurotrophic factor (BDNF) gene into a brain region called the hypothalamus improved metabolism in mice showing some symptoms of Prader-Willi syndrome (PWS), according to a new study. The gene therapy improvements translated into less weight gain and a healthier body composition, which suggests…

Daily treatment with oral pitolisant reduced excessive daytime sleepiness in children and adults with Prader-Willi syndrome (PWS), according to top-line data from a placebo-controlled Phase 2 clinical trial. While the relatively small proof-of-concept study wasn’t powered to detect statistically significant differences, the observed improvements with pitolisant relative to…