Targeting an enzyme called EHMT2 may help treat Prader-Willi syndrome (PWS) by reactivating the maternal PWS genes that are normally turned off, according to a new study. PWS is caused by the loss of certain genes in a region of the paternal chromosome 15 called the PWS locus.
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…
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…
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…
The U.S. Food and Drug Administration (FDA) has delayed by three months its decision on whether to approve Soleno Therapeutics’ extended-release formulation of diazoxide choline — known as DCCR — for curbing excessive hunger in people with Prader-Willi syndrome (PWS). The therapy candidate is designed for use in…
Hospitalizations among people with Prader-Willi syndrome (PWS) are characterized by higher healthcare utilization and complexity compared with non-PWS patients, according to a large-scale analysis of a U.S.-based administrative claims database. In addition to high rates of obesity, PWS patients had longer hospital stays, higher associated costs, and more in-hospital…
Neuren Pharmaceuticals has opened the first site for a U.S.-based Phase 2 clinical trial of its investigational therapy NNZ-2591 in children with Prader-Willi syndrome (PWS). “The Neuren team is very excited to be working with the community to complete this important first study of NNZ-2591 in young children…
EMO-T, a new emotional skill training program, helped children with Prader-Willi syndrome (PWS) express, recognize, understand, and regulate emotions, a study suggested. After EMO-T, scores related to emotional expression and comprehension matched those of control participants without PWS. Regulating and expressing emotions requires prerequisite emotion skills, said the researchers,…
Reduced activity of the Magel2 gene, which is defective in Prader-Willi syndrome (PWS), specifically in nerve cells that supply the brain’s amygdala, lowered the susceptibility to diet-induced weight gain by increasing physical activity. The researchers noted that these unexpected findings from a mouse study may provide a novel therapeutic strategy…
A Phase 2 study evaluating ARD-101, an investigational, gut-targeting Prader-Willi syndrome (PWS) oral therapy, is now enrolling up to 12 patients, ages 17 to 65. The open-label study, sponsored by Aardvark Therapeutics, the treatment’s developer, will be held at two sites: Children’s Hospital in Colorado, which is…
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