News

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…

After discontinuing Vykat XR (diazoxide choline) for a few months as part of a clinical trial, Prader-Willi syndrome (PWS) patients who restarted the treatment showed sustained reductions in insatiable hunger, or hyperphagia, and behavioral problems. The results were presented in a poster, titled “Resuming diazoxide choline extended-release (DCCR)…

Thanks to strong enrollment in a Phase 3 clinical trial of Acadia Pharmaceuticals’ ACP-101 — an experimental nasal spray of carbetocin for treating hyperphagia in Prader-Willi syndrome (PWS) — the company announced that it now expects to have top-line data later this year. That trial, COMPASS PWS…

Parents and siblings of people with Prader-Willi syndrome (PWS) commonly experience stress and other mental health challenges, but greater family cohesion, or bonding, helped buffer against these adverse outcomes, according to a study that surveyed people from around the world. In questionnaires completed by parents, parental depression, anxiety, and…

May is Prader-Willi Syndrome (PWS) Awareness Month and the worldwide PWS community is coming together to spread awareness about the rare genetic condition. Also, May 15 is PWS Awareness Day in the U.S. The Prader-Willi Syndrome Association USA (PWSA USA) is encouraging the community to engage in…