A newly published study on the use of guanfacine extended release (GXR) in individuals with Prader-Willi syndrome (PWS) marks an important step forward in addressing challenging behaviors such as aggression, skin-picking, and hyperactivity. Funded by FPWR, this was the first randomized, placebo-controlled clinical trial to evaluate a medication specifically for these neuropsychiatric symptoms in PWS.
The results have been published here in the American Journal of Medical Genetics Part B: Neuropsychiatric Genetics.
What Was Studied?
The study focused on guanfacine extended release (GXR), a medication sometimes prescribed to treat ADHD and other behavioral conditions. Researchers asked:
Could GXR help reduce some of the most difficult behaviors in PWS—specifically aggression, skin-picking, and hyperactivity?
Who Participated?
The trial included children and adults with PWS, ages 6–35. Participants were randomly assigned to receive either GXR or a placebo for a set period.
Importantly, neither participants nor researchers knew who was receiving the active medication during the trial—a method known as double-blind testing.
What Were the Results?
-
GXR significantly reduced aggression, skin-picking, and hyperactivity compared to placebo.
-
No serious side effects were reported. The most common side effect in the GXR group was mild, manageable sedation (feeling sleepy).
-
The medication was well-tolerated overall.
These results strongly support the potential of GXR as a safe and effective treatment for some of the most challenging neuropsychiatric symptoms in PWS.
Why This Matters
This is the first controlled clinical trial to show that a medication can safely and effectively target neuropsychiatric symptoms in PWS beyond food-related behaviors.
For many families, managing aggressive behavior and self-injury—like skin-picking—can be exhausting and emotionally draining. These new findings offer hope and evidence that GXR may become a meaningful option to ease some of those challenges.
A Heartfelt Thank You
This groundbreaking research was led by Dr. Deepan Singh, a clinician and researcher committed to improving psychiatric care for individuals with PWS. FPWR extends our deepest gratitude to Dr. Singh and the incredible families who participated in the study.
To our FPWR community: your generous support made this study possible. Thank you for continuing to drive progress in PWS research and care.
