Patricia Inácio, PhD,  science writer—

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to CSTI-500, Consynance Therapeutics’ oral treatment candidate for children and adolescents with Prader-Willi syndrome (PWS). That status is given to experimental therapies with the potential to provide clinically meaningful benefits to patients, primarily those younger than…

Adults with Prader-Willi syndrome (PWS) who were given growth hormone (GH) therapy in childhood are more likely to continue to be monitored by a multidisciplinary care team, according to results from a survey conducted in Japan. “These results emphasize the importance of the seamless provision of the multidisciplinary team…

Metabolic and bariatric surgery (MBS) can safely lead to sustained weight loss in Prader-Willi syndrome (PWS) patients, a meta-analysis shows. “It may indeed be time to take a new look at MBS for patients with PWS,” the researchers wrote. The study, “Metabolic and bariatric surgery for obesity…

Treatment with liraglutide along with an exercise and diet program eased excessive hunger in children and adolescents with Prader-Willi syndrome (PWS), but had no effects on body mass index (BMI) and other weight-related measures, according to the findings of a Phase 3 trial. The study, “Liraglutide…

Sleep disturbances in people with Prader-Willi syndrome (PWS) are linked with psychosis-risk symptoms, a study reports. Psychosis refers to disturbed thoughts and perceptions that may make it difficult to understand what is real and what’s not. The findings also indicate neurobehavioral symptoms and cognition can be analyzed remotely in…

A total of 30 patient advocacy groups working to further understanding and the needs of people living with rare diseases have been selected to receive a Horizon Therapeutics‘ #RAREis Global Advocate Grant. Winners of this year’s inaugural awards are spread across nine countries and represent a total of 29 rare…

Early treatment with growth hormone — before 1 year of age — was associated with better body composition and cognitive function after eight years in children with Prader-Willi syndrome (PWS), according to a recent Dutch study. The findings support additional research favoring early treatment with growth hormone for…

Saniona will pause the Phase 2b clinical trials testing Tesomet for people with Prader-Willi syndrome (PWS) and hypothalamic obesity (HO). The decision “is not related to the safety or efficacy of Tesomet and is entirely due to funding limitations,” the company stated in a press release. The…

The U.S. Food and Drug Administration (FDA) has told Levo Therapeutics that an additional clinical trial will be necessary to confirm the efficacy of LV-101 (intranasal carbetocin), a nasal spray aimed at reducing insatiable hunger, called hyperphagia, in Prader-Willi syndrome (PWS) patients. Levo Therapeutics had submitted a new drug…

A new study is looking for participants to assess the impact of a behavioral training program for caregivers of children with Prader-Willi syndrome (PWS). The training program, led by Kasey Bedard, PhD, at the Chicago School of Professional Psychology, will be conducted virtually by Zoom and Qualtrics, according to…