Parkinson's disease (PD), affecting 10 million people globally, lacks a cure, and current
therapies only manage symptoms. A link between Gaucher disease (GD) and PD, particularly in
carriers of glucocerebrosidase (GBA1) mutations, has sparked interest in developing new
drugs. Despite pharmaceutical companies focusing on formulations, progress is slow. Agyany,
with decades of experience in GD research, plans clinical trials using existing generic drugs
for GBA-related PD and idiopathic PD. Their approach targets the misfolded enzyme
glucocerebrosidase with pharmacological chaperons, inspired by success in GD using ambroxol.
The strategy aims to provide a quicker path to novel therapeutic options for PD.