Disease-Modifying Therapies in Parkinson's Disease.

A "disease-modifying" treatment is defined as an intervention capable of slowing the progressive worsening of clinical symptoms of a neurodegenerative disease. Over the last couple of decades, many efforts have been spent on disease-modifying compounds development in Parkinson's disease (PD). Herein, the authors present an overview of recent and ongoing clinical trials on PD disease-modifying therapies, stratified by mechanism of actions. Overall, several trials have been run to target alpha-synuclein in different ways, with disappointing results so far, despite preclinical positive findings. In the next few years, results on precision trials targeting genetic population or pathways (as β-glucocerebrosidase [GBA] or leucine-rich repeat kinase 2 mutations) are expected.