News

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…

The first eligible Prader-Willi syndrome (PWS) patients in the U.S. have received their prescriptions for Vykat XR (diazoxide choline) — the once-daily, extended-release tablets now approved as a treatment for hyperphagia, or insatiable hunger, in people with PWS, ages 4 and older. Soleno Therapeutics’ Vykat XR became…

The U.S. Food and Drug Administration (FDA) has approved Soleno Therapeutics’ extended-release formulation of diazoxide choline (DCCR) for easing hyperphagia — marked by insatiable hunger — in Prader-Willi syndrome (PWS) patients ages 4 and older. The newly authorized oral therapy will be marketed under the brand name…

Researchers have successfully used the gene-editing tool CRISPR to reactivate certain maternally-inherited genes that can compensate for the loss of the corresponding paternally-inherited genes in Prader-Willi syndrome (PWS). Done using lab-grown human stem cells with a PWS-causing mutation, the work showed that the one-time approach resulted in a sustained…

Rhythm Pharmaceuticals plans to start a new Phase 2 clinical study in the first quarter of 2025 to test setmelanotide in adults and children with Prader-Willi syndrome (PWS). An investigational therapy for PWS, setmelanotide is approved in the U.S. to treat certain types of genetic obesity under the…