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Exenatide once weekly over 2 years as a potential disease-modifying treatment for Parkinson’s disease: protocol for a multicentre, randomised, double blind, parallel group, placebo controlled, phase 3 trial: The ‘Exenatide-PD3’ study

Abstract:
Parkinson’s disease is the second most common neurodegenerative disease. Mitochondrial dysfunction has been associated with neurodegeneration in Parkinson’s disease, and several treatments targeting mitochondria have been tested in these patients to delay disease progression and tackle disease symptoms. Herein, we review available data from randomised, double-blind clinical studies that have investigated the role of compounds targeting mitochondria in idiopathic Parkinson’s disease patients, with a view of providing patients and clinicians with a comprehensive and practical paper that can inform therapeutic interventions in this group of people. A total of 9 compounds have been tested in randomized clinical trials, but only exenatide has shown some promising neuroprotective and symptomatic effects. However, whether this evidence can be translated into daily clinical practice still needs to be confirmed. In conclusion, targeting mitochondrial dysfunction in Parkinson’s disease is a promising therapeutic approach, although only one compound has shown a positive effect on Parkinson’s disease progression and symptoms. New compounds have been investigated in animal models, and their efficacy needs to be confirmed in humans through robust, randomised, double-blind clinical trials
Publication status:
Published
Peer review status:
Peer reviewed

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Publisher copy:
10.1136/bmjopen-2020-047993
Publication website:
https://discovery.ucl.ac.uk/10168160/1/37005519CNSNDDT-OA.pdf

Authors

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Role:
Author
ORCID:
0000-0002-9671-0212
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Role:
Author
ORCID:
0000-0001-5019-6812
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Role:
Author
ORCID:
0000-0003-2965-1870
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Role:
Author
ORCID:
0000-0003-0686-3053


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Funder identifier:
10.13039/501100007602
Grant:
16/167/19
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Grant:
BY-TF021 (DATScan substudy)


Publisher:
BMJ Publishing Group
Journal:
BMJ Open More from this journal
Volume:
11
Issue:
5
Pages:
e047993-e047993
Publication date:
2021-05-28
Acceptance date:
2021-05-12
DOI:
EISSN:
2044-6055
ISSN:
2044-6055


Language:
English
Keywords:
Pubs id:
1182723
Local pid:
pubs:1182723
Source identifiers:
W3164071625
Deposit date:
2026-03-24
ARK identifier:
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