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Ann Neurol. 2005 Feb;57(2):298-302.

Intraputamenal infusion of glial cell line-derived neurotrophic factor in PD: a two-year outcome study.

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Frenchay Hospital, Institute of Neurosciences, Bristol, United Kingdom.


We have shown previously that intraparenchymal infusion of glial cell line-derived neurotrophic factor (GDNF) continuously into the posterior putamen in five Parkinson's disease patients is safe and may represent a new treatment option. Here, we report a continuation of this phase I study. After 2 years of continual GDNF infusion, there were no serious clinical side effects and no significant detrimental effects on cognition. Patients showed a 57% and 63% improvement in their off-medication motor and activities of daily living subscores of the Unified Parkinson's Disease Rating Scale, respectively, and health-related quality-of-life measures (Parkinson's Disease Questionnaire-39 and Short Form-36) showed general improvement over time.

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