Precision Medicine for Parkinson's Disease: Ambroxol for Glucocerebrosidase-Associated Parkinson's Disease, First Trial Completed
- PMID: 32379375
- DOI: 10.1002/mds.28072
Precision Medicine for Parkinson's Disease: Ambroxol for Glucocerebrosidase-Associated Parkinson's Disease, First Trial Completed
Comment on
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Ambroxol for the Treatment of Patients With Parkinson Disease With and Without Glucocerebrosidase Gene Mutations: A Nonrandomized, Noncontrolled Trial.JAMA Neurol. 2020 Apr 1;77(4):427-434. doi: 10.1001/jamaneurol.2019.4611. JAMA Neurol. 2020. PMID: 31930374 Free PMC article. Clinical Trial.
References
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- Maegawa GH, Tropak MB, Buttner JD, et al. Identification and characterization of ambroxol as an enzyme enhancement agent for Gaucher disease. J Biol Chem 2009;284:23502-23516.
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- McNeill A, Magalhaes J, Shen C, et al. Ambroxol improves lysosomal biochemistry in glucocerebrosidase mutation-linked Parkinson disease cells. Brain 2014;137:1481-1495.
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- Migdalska-Richards A, Ko WKD, Li Q, Bezard E, Schapira AHV. Oral ambroxol increases brain glucocerebrosidase activity in a nonhuman primate. Synapse 2017;71. https://doi.org/10.1002/syn.21967
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- Mullin S, Smith L, Lee K, et al. Ambroxol for the treatment of patients with Parkinson disease with and without glucocerebrosidase gene mutations: a nonrandomized, noncontrolled trial [published online ahead of print January 13, 2020]. JAMA Neurol. https://doi.org/10.1001/jamaneurol.2019.4611
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