News

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 has opened the first site for a U.S.-based Phase 2 clinical trial of its investigational therapy NNZ-2591 in children with Prader-Willi syndrome (PWS). “The Neuren team is very excited to be working with the community to complete this important first study of NNZ-2591 in young children…

CSTI-500, an experimental oral therapy that ConSynance Therapeutics is developing to ease symptoms of Prader-Willi syndrome (PWS), showed a favorable safety and pharmacological profile in people with the disease, according to data from a Phase 1 clinical trial. “CSTI-500 has the potential to significantly improve the lives of…

Prader-Willi syndrome (PWS) Awareness Month happens every May to celebrate the global PWS community and call attention to its needs and there are a host of ways to participate this year. The goal is to heighten PWS awareness among lawmakers, scientists, public authorities, health professionals, and the general…

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…

Children younger than 2 with Prader-Willi syndrome (PWS) show no changes in the levels of free thyroxine (FT4) — the main hormone produced by the thyroid gland — following growth hormone treatment, a Japanese study shows. In older children, those ranging in age from 2 to 7, growth hormone…

The case of a young man with Prader-Willi syndrome who developed Hoffman’s syndrome, a condition marked by muscle weakness due to reduced thyroid gland function, was described in a recent report. The patient’s muscle weakness was effectively treated with thyroid hormone replacement. The study, “Hoffman’s syndrome as the…

Children with Prader-Willi syndrome (PWS) who start on treatment with growth hormone in the first year of life tend to have more normal growth parameters like height and weight, a new study reports. “This longitudinal study showed that younger children with PWS may benefit greatly from [growth hormone] treatment…

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…