Alice Melão,  —

A genetic analysis of hibernating animals and people with Prader-Willi syndrome (PWS) has revealed more than 300 small genetic sequences that may regulate fat accumulation and obesity, a study reports. This discovery adds new knowledge on the underlying processes of fat buildup and reveals new clues on potential genetic factors…

The ongoing DESTINY PWS Phase 3 trial is halfway to its target enrollment of patients with Prader-Willi syndrome (PWS) to evaluate the impact of once-daily treatment with diazoxide choline controlled-release (DCCR) tablets. Importantly, more than 90% of the patients who have completed the study so far have chosen to…

Treatment with once daily guanfacine extended-release tablets — used to treat attention deficit hyperactivity disorder (ADHD) — may help manage behavioral symptoms, such as aggression and self-injurious behavior, associated with Prader-Willi syndrome (PWS). The findings were reported in the study “Guanfacine Extended Release for…

Many patients with Prader-Willi syndrome may manifest psychotic symptoms that meet the diagnostic criteria of cycloid psychosis, a study shows. However, this particular psychological condition is often disregarded by clinicians, which may prevent prompt and adequate care. This finding was reported in a study, “…

A compound that could mimic ghrelin hormone activity and trigger its receptor signals could hold therapeutic potential to improve survival of patients with Prader-Willi syndrome (PWS) in early infancy, a mouse study shows. The study, “Ghrelin Receptor Agonist Rescues Excess…

Preclinical studies on the long-term impact of the investigational therapy ZGN-1258, being developed as a potential therapy for Prader-Willi syndrome (PWS), revealed alarming toxicity events and safety issues, Zafgen, the therapy’s developer, has announced. Based on these negative results, the company has changed its plans for ZGN-1258,…

Data from an ongoing Phase 2a clinical trial showed that Tesomet (tesofensine/metoprolol) is safe and well-tolerated by adolescents with Prader-Willi syndrome (PWS). Eight of the nine adolescents included in the trial have enrolled in an open-label extension (OE) study, where they will proceed with Tesomet treatment for an additional…

The U.S. Food and Drug Administration has granted an orphan drug designation to Auris Medical for its intranasal betahistine product for the treatment of obesity associated with Prader-Willi syndrome. In addition to Prader-Willi syndrome, the company also has plans to expand the betahistine development program to include depression…

Aripiprazole, a medication used to treat the symptoms of schizophrenia and other psychotic disorders, is a potential therapeutic option for patients with Prader-Willi syndrome and psychosis, a new case report suggests. The study, “Clinical Usefulness of Aripiprazole Treatment in a Girl…

Treatment with Soleno Therapeutics’ diazoxide choline controlled release (DCCR) tablets reduced excessive appetite and lowered body fat in Prader-Willi syndrome (PWS) patients in a Phase 2 trial, the company announced. The latest findings were presented during the Obesity Society Meeting 2018 in Nashville, Tennessee, in the poster, “…