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

  1. Schneider, Susanne A.
  2. Alcalay, Roy N.

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1000 Titel
  • Precision medicine in Parkinson’s disease: emerging treatments for genetic Parkinson’s disease
1000 Autor/in
  1. Schneider, Susanne A. |
  2. Alcalay, Roy N. |
1000 Erscheinungsjahr 2020
1000 Publikationstyp
  1. Artikel |
1000 Online veröffentlicht
  • 2020-01-23
1000 Erschienen in
1000 Quellenangabe
  • 267(3):860-869
1000 Copyrightjahr
  • 2020
1000 Lizenz
1000 Verlagsversion
  • https://doi.org/10.1007/s00415-020-09705-7 |
  • https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7035220/ |
1000 Publikationsstatus
1000 Begutachtungsstatus
1000 Sprache der Publikation
1000 Abstract/Summary
  • 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.
1000 Sacherschließung
lokal Genetic Predisposition to Disease [MeSH]
lokal TORC1 inhibitor
lokal Mutation [MeSH]
lokal Kinase inhibitor
lokal Humans [MeSH]
lokal Neurological Update
lokal SNCA
lokal Precision Medicine/methods [MeSH]
lokal Parkinson Disease/therapy [MeSH]
lokal GBA
lokal Genetic Parkinson’s disease
lokal Superprecision medicine
lokal Parkinson Disease/genetics [MeSH]
lokal Leucine-Rich Repeat Serine-Threonine Protein Kinase-2/genetics [MeSH]
lokal Glucosylceramidase/genetics [MeSH]
lokal Clinical Trials as Topic [MeSH]
lokal Clinical trial
lokal Small molecule compounds
lokal LRRK2
lokal Molecular Targeted Therapy/methods [MeSH]
lokal Ambroxol
lokal Venglustat
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1000 Erstellt am 2023-11-18T00:27:50.242+0100
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