News

Early findings from a Phase 2 clinical trial testing Rhythm Pharmaceuticals’ setmelanotide in children and adults with obesity due to Prader-Willi syndrome (PWS) are expected this year. That’s according to an update from Rhythm, which announced in a company press release that it plans to “disclose preliminary…

A Phase 3 trial testing the ability of Aardvark Therapeutics’ oral treatment candidate ARD-101 to ease hyperphagia, or insatiable hunger, in people with Prader-Willi syndrome (PWS) is still seeking adolescent and adult participants in the U.S., with other sites planned globally. Called HERO (NCT06828861), the study is…

Nearly all children with Prader-Willi syndrome (PWS) are hospitalized during their first year of life, a study reports. Hospitalizations in early childhood are generally more common with PWS than Angleman syndrome, another rare genetic disorder, the study found. Mortality rates also were higher in PWS, and 6% of…

People with Prader-Willi Syndrome (PWS) experience worse depression, overall health, and life satisfaction compared with the general population, according to validated questionnaires completed by more than 500 parents. With older age, depression tended to increase, while global health and life satisfaction tended to decrease, the data showed. “The validated…

A year of treatment with somatropin safely maintains or improves body composition in Japanese children and adults with Prader-Willi syndrome (PWS). The findings come from a Pfizer-funded Phase 3 clinical trial (NCT04697381) that evaluated somatropin’s one-year safety and efficacy in 33 PWS patients on stable growth hormone treatment…

Most children and adolescents with Prader-Willi syndrome (PWS) experience frequent anxiety and engage in distress-related behaviors that become a regular part of daily life for their families. That’s according to results from a survey completed by caregivers at the start of the Phase 3 CARE-PWS clinical trial (NCT03649477),…

Vykat XR (diazoxide choline) can safely reduce hyperphagia — a feeling of insatiable hunger that is a hallmark of Prader-Willi syndrome (PWS) — in patients whose access to food has been restricted and in those with pre-diabetes or diabetes. These data were presented in two poster presentations at…

Adenotonsillectomy, or surgery to remove the adenoids and tonsils, is less effective at relieving obstructive sleep apnea in children with Prader-Willi syndrome (PWS) than in those without the disease, according to a study in the U.S. Data also showed hormone therapies commonly used to manage PWS may further increase…

Different types of mutations that cause Prader-Willi syndrome (PWS) are associated with distinct patterns of changes in the brain and psychiatric disorders, a review study highlights. “This state-of-the-art review integrates cutting-edge findings in genomics, neuroimaging, patient-derived neuronal models, and computational analytics, thus providing the most comprehensive synthesis available to…

The extended-release formulation of diazoxide choline (DCCR) — approved in the U.S. earlier this year for use in Prader-Willi syndrome (PWS) — is now under review in the European Union as a treatment for hyperphagia, or insatiable hunger, in PWS patients ages 4 and older, according to its…