Authors
Anthony E Lang, Steven Gill, Nik K Patel, Andres Lozano, John G Nutt, Richard Penn, David J Brooks, Gary Hotton, Elena Moro, Peter Heywood, Matthew A Brodsky, Kim Burchiel, Patrick Kelly, Arif Dalvi, Burton Scott, Mark Stacy, Dennis Turner, VG Frederich Wooten, William J Elias, Edward R Laws, Vijay Dhawan, A Jon Stoessl, James Matcham, Robert J Coffey, Michael Traub
Publication date
2006/3
Journal
Annals of neurology
Volume
59
Issue
3
Pages
459-466
Publisher
Wiley Subscription Services, Inc., A Wiley Company
Description
Objective
Glial cell line–derived neurotrophic factor (GDNF) exerts potent trophic influence on midbrain dopaminergic neurons. This randomized controlled clinical trial was designed to confirm initial clinical benefits observed in a small, open‐label trial using intraputamenal (Ipu) infusion of recombinant human GDNF (liatermin).
Methods
Thirty‐four PD patients were randomized 1 to 1 to receive bilateral continuous Ipu infusion of liatermin 15μg/putamen/day or placebo. The primary end point was the change in Unified Parkinson Disease Rating Scale (UPDRS) motor score in the practically defined off condition at 6 months. Secondary end points included other UPDRS scores, motor tests, dyskinesia ratings, patient diaries, and 18F‐dopa uptake.
Results
At 6 months, mean percentage changes in “off” UPDRS motor score were −10.0% and −4.5% in the liatermin and placebo groups, respectively. This treatment …
Total citations
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