Marisa Wexler, MS, senior science writer —

N-Acetylcysteine (NAC) may help to reduce skin-picking behavior in some people with Prader-Willi syndrome (PWS), but many patients do not benefit from NAC treatment, a new study found. “NAC should continue to be considered as a treatment option for skin picking in PWS as over a third of severely…

A class of medications called GLP-1 receptor agonists (GLP1-RA) may help reduce appetite and promote weight loss for people with Prader-Willi syndrome (PWS), according to a review paper. The paper’s authors called for clinical trials to test these medicines in people with PWS. Their paper, “The…

Excessive eating in Prader-Willi syndrome (PWS) increases the risk of mortality not only by making patients prone to obesity and its associated health problems, but also by raising the risk of accidents like choking, a new study indicates. “There are the longer-term problems that arise through obesity and the…

During pregnancy, Prader-Willi syndrome (PWS) may manifest as an excess of fluid around the developing fetus coupled with restricted fetal growth and decreased fetal movement, a new report indicates. These signs should alert clinicians to consider testing for the possibility of a PWS diagnosis, the researchers wrote. The…

People with Prader-Willi syndrome (PWS) have smaller pituitary glands than their typically-developing peers, according to data from a small study. The findings also suggest that altered pituitary size is associated with behavioral abnormalities in PWS. Results were published in the journal Biomarkers in Neuropsychiatry, in the study “…

Aardvark Therapeutics has raised $29 million in financing to help support three planned Phase 2 clinical trials of its lead compound, ARD-101, an investigational therapy for Prader-Willi syndrome (PWS) and other disorders. Financing was led by Sorrento Therapeutics, with participation from other investors and support from the…

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…

People with Prader-Willi syndrome (PWS) and other diseases commonly experience sleep problems, and the problems are rarely addressed effectively by healthcare professionals, according to a white paper published by Harmony Biosciences. Excessive daytime sleepiness, or EDS, is a common symptom experienced by people with rare diseases…

Specific genetic subtypes and growth hormone use both influence the physical characteristics, such as head shape and eye appearance, of people with Prader-Willi syndrome (PWS), a large study indicates. The study, “Influence of Molecular Classes and Growth Hormone Treatment on Growth and Dysmorphology in Prader‐Willi Syndrome:…

Soleno Therapeutics has partnered with Vanderbilt University to develop therapies that work as ATP-dependent potassium channel activators for rare diseases, similar to the diazoxide choline controlled release (DCCR) tablets the company thinks might treat Prader-Willi syndrome (PWS). ATP-dependent potassium (or KATP) channels are proteins present in multiple body tissues.