Cough medicine repurposed for rare brain disease in kids

This study tested high doses of Ambroxol, a common cough medicine, in 12 children aged 3 to 18 with Type 3 Gaucher disease, a rare genetic disorder affecting the brain and body. The goal was to see if it is safe and can improve movement problems like ataxia. The trial took place in Lahore, Pakistan, and included children who were either new to treatment or already on enzyme therapy.