Targeted Therapies for Parkinson's Disease: From Genetics to the Clinic

Mov Disord. 2018 May;33(5):684-696. doi: 10.1002/mds.27414. Epub 2018 Apr 27.


The greatest unmet medical need in Parkinson's disease (PD) is treatments that slow the relentless progression of symptoms. The discovery of genetic variants causing and/or increasing the risk for PD has provided the field with a new arsenal of potential therapies ready to be tested in clinical trials. We highlight 3 of the genetic discoveries (α-synuclein, glucocerebrosidase, and leucine-rich repeat kinase) that have prompted new therapeutic approaches now entering the clinical stages. We are at an exciting juncture in the journey to developing disease-modifying treatments based on knowledge of PD genetics and pathology. This review focuses on therapeutic paradigms that are under clinical development and highlights a wide range of key outstanding questions in PD. © 2018 The Authors. Movement Disorders published by Wiley Periodicals, Inc. on behalf of International Parkinson and Movement Disorder Society.

Keywords: GBA LRRK2; SNCA; clinical trial; glucocerebrosidase; α-synuclein.

Publication types

  • Review

MeSH terms

  • Animals
  • Clinical Trials as Topic / methods*
  • Glucosylceramidase / genetics
  • Humans
  • Parkinson Disease / genetics*
  • Parkinson Disease / therapy*
  • Protein Serine-Threonine Kinases / genetics
  • alpha-Synuclein / genetics


  • alpha-Synuclein
  • Protein Serine-Threonine Kinases
  • Glucosylceramidase