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J Neurosci. 2018 Oct 31;38(44):9375-9382. doi: 10.1523/JNEUROSCI.1666-18.2018.

New Frontiers in Parkinson's Disease: From Genetics to the Clinic.

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Neuroscience Therapeutic Area, Sanofi, Framingham, MA 01701,
Van Andel Research Institute, Grand Rapids, MI 49503.
Pittsburgh Institute for Neurodegenerative Diseases, University of Pittsburgh, Pittsburgh, PA 15260, and.
Critical Path Institute, CAMD, Tucson, AZ 85718.
Neuroscience Therapeutic Area, Sanofi, Framingham, MA 01701.


The greatest unmet therapeutic need in Parkinson's disease (PD) is a treatment that slows the relentless progression of the symptoms and the neurodegenerative process. This review highlights the utility of genetics to understand the pathogenic mechanisms and develop novel therapeutic approaches for PD. The focus is on strategies provided by genetic studies: notably via the reduction and clearance of α-synuclein, inhibition of LRRK2 kinase activity, and modulation of glucocerebrosidase-related substrates. In addition, the critical role of precompetitive public-private partnerships in supporting trial design optimization, overall drug development, and regulatory approvals is illustrated. With these great advances, the promise of developing transformative therapies that halt or slow disease progression is a tangible goal.


GBA; LRRK2; Parkinson's disease; alpha-synuclein; disease-modifying therapies

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