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Acta Pharm Sin B (2016) 6(5):41325

Furthermore, BDNF has never entered PD clinical trials, mainly due to poor blood-brain barrier penetration, a short half-life, and the risk of abnormal synaptic plasticity (e.g., epileptiform discharges) with overexpression [62, 63]

These factors promote the activation of fibroblasts and the excessive production of ECM components

Current therapies include ERT to compensate for the missing or defective enzyme, and substrate reduction therapy to reduce the accumulation of toxic substances by limiting their production