Lindsey Shapiro, PhD,  science writer—

A Phase 3 trial testing the ability of Aardvark Therapeutics’ oral treatment candidate ARD-101 to ease hyperphagia, or insatiable hunger, in people with Prader-Willi syndrome (PWS) is still seeking adolescent and adult participants in the U.S., with other sites planned globally. Called HERO (NCT06828861), the study is…

Parents and siblings of people with Prader-Willi syndrome (PWS) commonly experience stress and other mental health challenges, but greater family cohesion, or bonding, helped buffer against these adverse outcomes, according to a study that surveyed people from around the world. In questionnaires completed by parents, parental depression, anxiety, and…

Throughout May, supporters will celebrate Prader-Willi Syndrome (PWS) Awareness Month with a variety of educational, advocacy, and fundraising initiatives. The goal of the month-long event is to raise awareness of the rare genetic disease and to support efforts that will accelerate PWS research and treatment development. As it has…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Harmony Biosciences’ pitolisant to treat daytime sleepiness in people with Prader-Willi syndrome (PWS). Orphan drug status is meant to incentivize the development of treatments for rare diseases, or those affecting fewer than 200,000 people in…

While food-seeking behaviors normally lessen as people age, they increase over time with Prader-Willi syndrome (PWS), according to a study using the Hyperphagia Questionnaire for Clinical Trials (HQ-CT), a tool commonly used in clinical trials but not in comparing a large PWS patient group to the general population. HQ-CT…

Prader-Willi syndrome (PWS) patients who switched from daily treatment with DCCR (diazoxide choline extended-release tablets) to a placebo experienced worsening excessive hunger, or hyperphagia, and greater body weight gains relative to those who stayed on DCCR. That’s according to top-line data from the new randomized withdrawal period of…

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…