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Review
. 2020 Mar;267(3):860-869.
doi: 10.1007/s00415-020-09705-7. Epub 2020 Jan 23.

Precision medicine in Parkinson's disease: emerging treatments for genetic Parkinson's disease

Susanne A Schneider  1 , Roy N Alcalay  2
Affiliations
Review

Precision medicine in Parkinson's disease: emerging treatments for genetic Parkinson's disease

Susanne A Schneider et al. J Neurol. 2020 Mar.

Abstract

In recent years, numerous clinical trials for disease modification in Parkinson's disease (PD) have failed, possibly because of a "one-size-fits all" approach. Alternatively, a precision medicine approach, which customises treatments based on patients' individual genotype, may help reach disease modification. Here, we review clinical trials that target genetic forms of PD, i.e., GBA-associated and LRRK2-associated PD. In summary, six ongoing studies which explicitely recruit GBA-PD patients, and two studies which recruit LRRK2-PD patients, were identified. Available data on mechanisms of action, study design, and challenges of therapeutic trials are discussed.

Keywords: Ambroxol; Clinical trial; GBA; Genetic Parkinson’s disease; Kinase inhibitor; LRRK2; SNCA; Small molecule compounds; Superprecision medicine; TORC1 inhibitor; Venglustat.

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Conflict of interest statement

Dr. Alcalay reports receiving consultation fees from Genzyme/Sanofi, Roche and ResTORbio.

Figures

Fig. 1
Fig. 1
Treatment approaches for GBA-associated PD include modulation of gylcosphingolipid turnover and restoration of enzyme function
Fig. 2
Fig. 2
World map of LRRK2-associated Parkinsonism. 533 cases have been reported. Circles reflect frequency per region. Data and image were retrieved from the MDSGene Website [47]
See this image and copyright information in PMC

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