Lindsey Shapiro, PhD, science writer —

Harmony Biosciences plans to launch a Phase 3 clinical trial by year’s end to investigate whether pitolisant can safely and effectively reduce excessive daytime sleepiness in children, adolescents, and adults with Prader-Willi syndrome (PWS), the company said. The trial will be registrational, meaning that, if positive, the findings…

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…

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…

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…

The loss of certain genetic material called non-coding RNA might lead to the disruption of genes important for brain development and function in Prader-Willi syndrome (PWS) , a study found. These alterations could underlie the cognitive and behavioral symptoms of the disease,  according to researchers. The study, “…

A randomized withdrawal period in the ongoing open-label extension trial of DCCR (diazoxide choline extended-release tablets) for people with Prader-Willi syndrome (PWS) has been initiated to generate more data about the investigational treatment’s effectiveness. The study, called C602 (NCT03714373), is evaluating the safety and effectiveness of daily…

The first patient has been randomly assigned to a dosing group in the pivotal Phase 2/3 SCOUT-015 trial, which will evaluate the safety and efficacy of RAD011, an investigational cannabidiol oral treatment, in people with Prader-Willi syndrome (PWS). SCOUT-015 (NCT05098509) is designed to test the effects of…

A special low-calorie diet and appetite suppressing medications — either alone or together — led to substantial weight loss for obese people with Prader-Willi syndrome (PWS), a small study showed. However, many patients struggled to adhere to the treatment regimens and eventually regained the weight, the researchers reported. A very…

A year of treatment with DCCR (diazoxide choline controlled release) tablets, Soleno Therapeutics’ investigational therapy for people with Prader-Willi syndrome (PWS), safely led to sustained reductions in excessive hunger and disease-related behaviors, according to two presentations of data from a Phase 3 trial and its open-label extension. Soleno announced positive findings…

This year’s Prader-Willi syndrome (PWS) Awareness Month has launched, and will be celebrated with several educational and fundraising initiatives worldwide throughout May. Advocates will use the month-long event to raise awareness of the rare disease in the general community, and also among healthcare professionals, lawmakers, and researchers. The aim…