id	author	title	date	pages	extension	mime	words	sentences	flesch	summary	cache	txt
cord-286865-6imc98f5	Schneider, Susanne A.	Emerging Targeted Therapeutics for Genetic Subtypes of Parkinsonism	2020-09-10		.txt	text/plain	8315	439	45	Intervention on such mutations would require a Fig. 1 Genetic architecture of Parkinson's disease, modified from [4] and [5] , showing the continuum of variants of different effect strengths and allele frequencies. The G2019S mutation, for example, results in a direct two-to-threefold increase in kinase activity [33, 34] .The potential gain-of-function effect is an attractive target for treatment because inhibition is easier to achieve than improvement of reduced protein activity (as in GBA). The effects of LTI-291, an activator of the GCase enzyme and another small molecule therapy, were studied in a 1month phase 1b trial conducted in the Netherlands, where the frequency of GBA mutations was reported to be around 15% [48] . Human gene therapy approaches for the treatment of Parkinson's disease: An overview of current and completed clinical trials Ambroxol for the Treatment of Patients With Parkinson Disease With and Without Glucocerebrosidase Gene Mutations: A Nonrandomized, Noncontrolled Trial	./cache/cord-286865-6imc98f5.txt	./txt/cord-286865-6imc98f5.txt