New Era in disease modification in Parkinson's disease: Review of genetically targeted therapeutics

S. Pablo Sardi, Tanya Simuni*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

14 Scopus citations


Disease modification remains a major unmet need in Parkinson's disease (PD) therapeutics. Despite multiple attempts, not a single study has yet been successful, perhaps due to our incomplete understanding of the underlying disease mechanisms. Genetic and epidemiologic studies of the last decade have substantially increased our comprehension of the etiology of PD. Once considered a pure sporadic disease, the discovery of familial mutations provided the initial paradigm shift and it is now widely accepted that PD has a substantial genetic component. These genetic discoveries have allowed the development of novel therapeutics aimed at halting or slowing the underlying disease process, rather than just ameliorating symptoms. Here, we discuss the latest advances in therapeutics based on three genetic discoveries (SNCA, LRRK2 and GBA) that are currently reaching the clinical arena and outline the challenges of therapeutic development of genetically targeted therapeutics.

Original languageEnglish (US)
Pages (from-to)32-38
Number of pages7
JournalParkinsonism and Related Disorders
StatePublished - Feb 2019


  • Clinical trials
  • Disease modification
  • Genetics
  • Parkinson's disease

ASJC Scopus subject areas

  • Neurology
  • Geriatrics and Gerontology
  • Clinical Neurology

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