Dietary Supplement Shows Promise in Angelman Syndrome Trial
A small clinical trial has found that a dietary supplement, designed to mimic a ketogenic diet, was well-tolerated by children with Angelman syndrome. The supplement, called ketogenic medical food formulation (KF), showed promising results with no negative effects on various aspects of the children's health.
The trial, conducted in four phases over 16 weeks, enrolled 14 children aged 4 to 11. Thirteen children completed the study, indicating good acceptance of the powder formulation. Almost all participants experienced softer stool consistency while on KF, a significant improvement noted by their families.
The supplement's efficacy could not be determined due to the small number of participants. However, it was not found to be harmful, with no negative impacts on cognition, seizure count, brain electrical activity, gait, mobility, sleep, digestive health, or developmental stage. Two adverse events were reported - elevated triglycerides and abnormally low albumin levels - but these were potentially related to the supplement or placebo. Families also reported reduced food-seeking behavior in some children while on the supplement.
The trial met its primary goal of assessing the supplement's tolerability. Half of the families chose to continue using the formula after the study concluded, suggesting a positive response to the supplement.
Biomond, the company that manufactured the supplement, and Disruptive Nutrition, which provided the KF, can take encouragement from these results. While the supplement's efficacy remains uncertain due to the small sample size, its tolerability and potential benefits warrant further investigation. Larger-scale studies are needed to confirm these initial findings and assess the long-term effects of the supplement on children with Angelman syndrome.
