Lindsey Shapiro, PhD,  science writer—

A randomized withdrawal period in the ongoing open-label extension trial of DCCR (diazoxide choline extended-release tablets) for people with Prader-Willi syndrome (PWS) has been initiated to generate more data about the investigational treatment’s effectiveness. The study, called C602 (NCT03714373), is evaluating the safety and effectiveness of daily…

The first patient has been randomly assigned to a dosing group in the pivotal Phase 2/3 SCOUT-015 trial, which will evaluate the safety and efficacy of RAD011, an investigational cannabidiol oral treatment, in people with Prader-Willi syndrome (PWS). SCOUT-015 (NCT05098509) is designed to test the effects of…

A special low-calorie diet and appetite suppressing medications — either alone or together — led to substantial weight loss for obese people with Prader-Willi syndrome (PWS), a small study showed. However, many patients struggled to adhere to the treatment regimens and eventually regained the weight, the researchers reported. A very…

A year of treatment with DCCR (diazoxide choline controlled release) tablets, Soleno Therapeutics’ investigational therapy for people with Prader-Willi syndrome (PWS), safely led to sustained reductions in excessive hunger and disease-related behaviors, according to two presentations of data from a Phase 3 trial and its open-label extension. Soleno announced positive findings…

This year’s Prader-Willi syndrome (PWS) Awareness Month has launched, and will be celebrated with several educational and fundraising initiatives worldwide throughout May. Advocates will use the month-long event to raise awareness of the rare disease in the general community, and also among healthcare professionals, lawmakers, and researchers. The aim…

Impaired social responsiveness, an indicator of autism spectrum disorder (ASD), is prevalent among adults with Prader-Willi syndrome (PWS), and is associated with worse social functioning, according to a recent study. “Our results suggest that evaluating difficulties in social skills is fundamental for the global assessment of functionality,” the researchers…

Soleno Therapeutics has announced that its diazoxide choline controlled release (DCCR) tablets will be evaluated in a clinical trial for people with early-phase Prader-Willi Syndrome (PWS), which will be funded by Running for Research – Prader-Willi Syndrome, an ongoing fundraiser to benefit PWS research operated by the University…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to TNX-2900, Tonix Pharmaceuticals’ oxytocin therapy for Prader-Willi syndrome (PWS). Orphan designation is intended to speed the development of potential therapies for rare diseases, or those affecting fewer than 200,000 people in the U.S. The status…

A case of acute kidney injury in a woman with Prader-Willi syndrome (PWS) and intellectual disability was attributed in a recent report to the use of multiple medications. The case shed lights on how vulnerable people with an intellectual disability are to being over-medicated, with the complications…

Several characteristics during pregnancy and immediately after birth — such as maternal age, pregnancy complications, low birth size, reduced muscle tone, and infant feeding difficulties — may distinguish a newborn with Prader-Willi Syndrome (PWS) from healthy babies early in life, a study showed. “By providing an overview of…