Author:
Schneider Susanne A.,Alcalay Roy N.
Abstract
AbstractIn 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.
Funder
Medizinischen Fakultät, Ludwig-Maximilians-Universität München
Publisher
Springer Science and Business Media LLC
Subject
Clinical Neurology,Neurology
Reference66 articles.
1. Dorsey ER, Elbaz A, Nichols E, Abd-Allah F, Abdelalim A, Adsuar JC et al (2018) Global, regional, and national burden of Parkinson’s disease, a systematic analysis for the global burden of disease study. Lancet Neurol 17:939–953
2. Abrahams E, President | personalized medicine coalition personalized medicine: The changing landscape of health care; key note lecture, The 2nd biomarker meeting in personalized reproductive medicine valencia, Spain [http://www.comtecmed.com/biomarker/2014/Uploads/Editor/PDF/ppt/Edward%20Abrahams_Key%20Note%20Lecture.pdf]. Accessed 16 Jan 2020
3. Collins FS, Varmus H (2015) A new initiative on precision medicine. N Engl J Med 372:793–795
4. Gasser T (2015) Usefulness of genetic testing in PD and PD trials: a balanced review. J Park Dis 5:209–215
5. Nalls MA, Pankratz N, Lill CM et al (2014) Large-scale meta-analysis of genome-wide association data identifies six new risk loci for Parkinson's disease. Nat Genet 46:989–993
Cited by
72 articles.
订阅此论文施引文献
订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献