Margarida Maia, PhD, science writer —

The U.S. Food and Drug Administration (FDA) will review Soleno Therapeutics’ application seeking approval of its extended-release formulation of diazoxide choline (DCCR) for Prader-Willi syndrome (PWS) in patients, 4 and older, with excessive hunger. The new drug application (NDA), filed in July, was granted priority review, shortening…

Soleno Therapeutics is seeking approval of diazoxide choline controlled-release (DCCR) tablets in the U.S. for Prader-Willi syndrome (PWS) patients, 4 and older, with excessive hunger. The filing of the new drug application with the U.S. Food and Drug Administration (FDA) follows positive data from an open-label extension…

About every 10th person with Prader-Willi syndrome (PWS) experiences epilepsy or fever-induced seizures, and more likely so if the disease is caused by a specific type of mutation, a review study has found. Also, this type of mutation — deletions in a segment of chromosome 15 and the most common…

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…