Families of children and adults with Prader-Willi Syndrome (PWS) know that challenges extend far beyond food-related behaviours. Many individuals with PWS also experience aggression, hyperactivity, impulsivity, and skin-picking—behaviours that can be incredibly difficult to manage at home, in school, and in the wider community.
But there’s encouraging news: a recent clinical trial has shown that a medication called Guanfacine Extended Release (GXR) may help reduce these behaviours safely and effectively.
What is Guanfacine Extended Release (GXR)?
GXR is a medication already approved to treat ADHD in children and teens. It works by calming specific areas of the brain that regulate attention, impulse control, and emotional responses. Because of these effects, researchers explored whether GXR could also help with the behavioural symptoms commonly seen in PWS.
About the Study
This research was funded by FPWR and led by Dr. Deepan Singh, a child and adolescent psychiatrist with extensive experience in PWS. The U.S.-based study involved 89 individuals with PWS, aged 6 to 35, who participated in a randomised, double-blind, placebo-controlled trial of GXR. This means that:
- Some participants received GXR
- Others received a placebo (a pill with no active ingredients)
- Neither the families nor the researchers knew who received which until the study concluded
Encouraging Results
The findings from this phase of the trial were very promising, showing that:
- Aggression and hyperactivity decreased significantly in those who took GXR compared to those on placebo
- Skin-picking, a common and distressing behaviour in PWS, also showed improvement
The medication was well tolerated, with no serious side effects reported. The most common side effect was mild sleepiness, which was generally manageable.
What Does This Mean for PWS?
This marks an exciting step forward in addressing some of the most challenging non-food-related behaviours in PWS. While the urgent need to treat hyperphagia remains a top priority, behaviours like aggression, impulsivity, and skin-picking can have a profound impact on school performance, relationships, and overall quality of life—for both individuals and their caregivers.
Having a safe and effective treatment option could be life changing.
What’s Next?
This study opens the door to further research aimed at:
- Understanding how GXR works specifically in people with PWS
- Determining the optimal dosage and treatment duration
- Exploring how GXR might be combined with other therapies for even better outcomes
Looking Ahead
This is a promising development in the treatment of some of the behavioural symptoms in PWS. We’re encouraged by the results and excited to see how this research evolves. FPWR UK will continue to follow this closely and keep our community updated as new findings emerge.
This study has been published in the American Journal of Medical Genetics Part B: Neuropsychiatric Genetics. which you can find at https://onlinelibrary.wiley.com/doi/10.1002/ajmg.b.33032