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

被引：67

作者：

Schneider, Susanne A. ^{[1
]}

Alcalay, Roy N. ^{[2
]}

机构：

[1] Ludwig Maximilians Univ Munchen, Dept Neurol, Marchioninistr 15, D-81377 Munich, Germany

[2] Columbia Univ, Dept Neurol, Med Ctr, New York, NY USA

来源：

JOURNAL OF NEUROLOGY | 2020年 / 267卷 / 03期

关键词：

Genetic Parkinson's disease; SNCA; GBA; LRRK2; Kinase inhibitor; Small molecule compounds; Venglustat; Ambroxol; Clinical trial; TORC1; inhibitor; Superprecision medicine; GAUCHER-DISEASE; GLUCOCEREBROSIDASE MUTATIONS; THERAPEUTIC STRATEGY; RISK LOCI; LRRK2; IDENTIFICATION; METAANALYSIS; ASSOCIATION; PENETRANCE; INHIBITORS;

D O I：

10.1007/s00415-020-09705-7

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

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.

引用

页码：860 / 869

页数：10

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