Publications by authors named "Abe Durrant"
J Clin Pharmacol
November 2024
Article Synopsis
- A study called ACTION-Galactosemia Kids evaluated the effects of govorestat, a drug for Classic Galactosemia, on children aged 2-17 by comparing it to a placebo over 18 months.
- Govorestat showed a significant and lasting reduction in plasma galactitol levels, leading to stabilization or improvement in various clinical measures like behavior and daily living skills, while the placebo group showed decline.
- Despite its benefits, govorestat did not show improvements in speech or gross motor skills, and both the drug and placebo groups experienced similar rates of adverse effects, indicating it is safe and well tolerated.
