Journal
JOURNAL OF NEUROLOGY
Volume 267, Issue 3, Pages 860-869Publisher
SPRINGER HEIDELBERG
DOI: 10.1007/s00415-020-09705-7
Keywords
Genetic Parkinson's disease; SNCA; GBA; LRRK2; Kinase inhibitor; Small molecule compounds; Venglustat; Ambroxol; Clinical trial; TORC1 inhibitor; Superprecision medicine
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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.
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