Marta Figueiredo, PhD,  Managing Science Editor—

A multidisciplinary approach that included weight reduction through calorie restriction and diuretics, ventilation support, skin care, and rehabilitation successfully managed life-threatening right heart failure due to severe obesity in a young man with Prader-Willi syndrome (PWS). These findings from a case study highlight not only the effectiveness of such a…

There is no substantial evidence supporting the efficacy of LV-101 (intranasal carbetocin), a nasal spray designed to reduce the insatiable hunger, called hyperphagia, that’s a hallmark of Prader-Willi syndrome (PWS), according to a 12-1 vote by a U.S. Food and Drug Administration (FDA) advisory committee. The overwhelmingly negative vote followed…

The Prader-Willi Syndrome Association (PWSA) USA announced that this year’s Angel Drive donation campaign — supporting the nonprofit in assisting those with this complex genetic disorder, and in advocating and funding research — will start on Nov. 1. With chapters in most U.S. states, PWSA USA is the only national…

The U.S. Food and Drug Administration (FDA) is holding a public advisory committee meeting regarding LV-101 (intranasal carbetocin) as a potential treatment of distress behaviors and insatiable hunger in Prader-Willi syndrome (PWS) patients. The Psychopharmacologic Drugs Advisory Committee meeting, to be held virtually Nov. 4 from 10…

One year of treatment with Soleno Therapeutics’ investigational oral therapy diazoxide choline controlled release, known as DCCR, significantly reduced excessive appetite and disease-related behaviors in Prader-Willi syndrome (PWS) patients, according to top-line data from an extension study of the DESTINY PWS Phase 3 trial. These trajectories were significantly…

Vision problems such as lazy eye, farsightedness, and strabismus — when the eyes don’t line up in the same direction — are more common in people with Prader-Willi syndrome (PWS) than among their healthy peers, a study shows. These findings, along with data showing that vision problems may negatively affect…

Children and adults with Prader-Willi syndrome (PWS) are not at higher risk of developing a severe COVID-19 infection, according to results from a survey involving more than 500 patients in France. These findings were particularly surprising in terms of the adult population, which typically has several known risk factors for severe…

Supervised exercise can improve physical fitness and increase lean body mass in people with Prader-Willi syndrome (PWS), and boost bone health in pediatric patients, according to a review study. These benefits occur even in the absence of weight and fat loss in these patients, who are less active than…

New brain-like organoids, or “mini-brains,” that are specific to key regions and derived from patients could serve as models for studies of the underlying neurological and molecular mechanisms of Prader-Willi syndrome (PWS), researchers reported. Notably, the team created arcuate organoids generated from cells of PWS patients, which captured the arcuate…

The Foundation for Prader-Willi Research (FPWR) and the Prader-Willi Syndrome Association (PWSA) in the U.S. have asked the U.S. Food and Drug Administration (FDA) to reconsider its request for an additional clinical trial of diazoxide choline controlled release (DCCR) tablets in patients with Prader-Willi syndrome (PWS). Last month, the…