News

Aardvark Therapeutics plans to expand its ongoing Phase 3 clinical trial of ARD-101, an oral therapy for easing excessive hunger in Prader-Willi syndrome (PWS), to include patients younger than 13. The decision, supported by the PWS community, is based on historical data indicating that younger PWS patients are…

Relmada Therapeutics is working toward the launch, next year, of a Phase 2 clinical trial to test sepranolone — which the company believes will be a first-in-class treatment for easing compulsive behaviors — in people with Prader-Willi syndrome (PWS). Preparations include engaging U.S. Food and Drug Administration officials…

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…