Margarida Maia, PhD, science writer —

Harmony Biosciences has started a pivotal Phase 3 clinical trial testing pitolisant for excessive daytime sleepiness (EDS) and behavioral problems in people with Prader-Willi syndrome (PWS), 6 and older. A pivotal study is one where results, if positive, are used to support applications for the therapy’s regulatory approval.

Acadia Pharmaceuticals has launched a Phase 3 clinical trial testing the safety and efficacy of ACP-101, an experimental carbetocin nasal spray, for the treatment of hyperphagia, or insatiable hunger, in people with Prader-Willi syndrome (PWS). The pivotal trial, dubbed COMPASS PWS, is expected to enroll up to…

Providing needed care for someone with Prader-Willi syndrome (PWS) may make people — particularly those who serve as primary caregivers — more likely to experience depression and feel overloaded, a small study from Argentina suggests. “Caring for people with PWS can have a significant effect on the mental health,…

Too much insulin — a hormone that controls sugar in blood — may set off weight gain and excessive eating (known as hyperphagia) in children with Prader-Willi syndrome (PWS), a study suggests. High levels of insulin occurred hand in hand with high levels of leptin, a hormone that controls…

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…

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…

Half of people with Prader-Willi syndrome (PWS) treated with the antipsychotic aripiprazole in a small German study experienced fewer temper outbursts, but daytime sleepiness was a common side effect. The medication did not cause an increase in weight, however, which is a plus considering that people with PWS typically…

Cells of adipose tissue, or body fat, may go awry long before weight gain takes off in children with Prader–Willi syndrome (PWS), according to a new study — and a missing cluster of genes called SNORD116 may be the reason why. The findings add information about what’s behind the…

There may be changes to how the “feel-good” hormone oxytocin works in patients with Prader-Willi syndrome (PWS) in general, and in those with psychosis, according to findings from a small study in Germany. Researchers found that OXTR, the gene that codes for the oxytocin receptor, is not as methylated in…

People with Prader-Willi syndrome (PWS) who had COVID-19 experienced either no symptoms or only mild illness due to the infection, an international survey study found. These findings were unexpected, as researchers had put forward the hypothesis that the presence of other conditions typically coexisting with PWS would make symptoms…