News

PWS-causing mutations tied to unique psychiatric disorder patterns

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…

Bonding helps ease family stress faced by PWS patients’ relatives

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…

Worldwide PWS community to make way for awareness month

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…

Hyperphagia treatment Vykat XR for PWS now available in US

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…

FDA approves DCCR, now Vykat XR, to ease hyperphagia in PWS

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…

Gene-editing tool successfully reactivates genes in PWS lab study

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…