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

ABSTRACT: 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 (?-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.

SUBMITTER: Sardi SP

PROVIDER: S-EPMC6282975 | biostudies-literature | 2018 May

REPOSITORIES: biostudies-literature

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

Sardi S Pablo SP Cedarbaum Jesse M JM Brundin Patrik P

Movement disorders : official journal of the Movement Disorder Society 20180427 5

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 a ...[more]

PMID: 29704272

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