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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…

Note: This story was updated July 13, 2022, to correct the name of Rare-X’s CEO Charlene Son Rigby. Nonprofits, scientists, governmental organizations, and the rare disease drug development industry have long cited 7,000 as the average number of rare diseases in the world.

Early treatment with growth hormone — before 1 year of age — was associated with better body composition and cognitive function after eight years in children with Prader-Willi syndrome (PWS), according to a recent Dutch study. The findings support additional research favoring early treatment with growth hormone for…

Testosterone injections are well-tolerated and normalize blood levels of the hormone, but they may raise red blood cell levels excessively in adult men with Prader-Willi syndrome (PWS), according to a recent study. The study, “Single-center real-life experience with testosterone treatment in adult men with Prader-Willi syndrome,”…

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…

The National Organization for Rare Disorders’ (NORD) “Living Rare, Living Stronger Patient and Family Forum” is back in person on June 26 for a day of learning and networking in Cleveland, Ohio. The event, which brings together the rare disease community, will take place at the InterContinental Cleveland Conference…

Adults with Prader-Willi syndrome (PWS) who weigh more, have a higher body mass index (BMI), and have a larger waist and hip circumference are most likely to respond successfully to a multidisciplinary weight reduction program, according to a single-center, six-year study in Italy. In addition, having PWS caused by…

Certain regions of the brain are unusually small in people with Prader-Willi syndrome (PWS), a study reveals. “The present study provides objective evidence of volumetric alterations in the subcortical, limbic, and brainstem structures in individuals with PWS,” its researchers wrote. The study, “Differential volume reductions in…

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…