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A Systematic Review on Disease-Modifying Therapies in Parkinsonian Disorders
Pepijn P N M Eijsvogel1,2, Lars M G Smits1,2, Philip H C Kremer1,2
1Centre for Human Drug Research, Leiden, The Netherlands.
Clinical Pharmacology and Therapeutics
|May 9, 2026
Summary
No disease-modifying therapies currently exist for Parkinsonian disorders. While some treatments show promise, including alpha-synuclein targeting and glucocerebrosidase enhancers, none have been approved, highlighting the need for better trial designs and biomarkers.
Area of Science:
- Neuroscience
- Pharmacology
- Clinical Neurology
Background:
- Parkinsonian disorders (Parkinson's disease, Lewy body dementia, multiple system atrophy, progressive supranuclear palsy) are neurodegenerative and lack disease-slowing treatments.
- Current therapeutic options do not halt the progression of these debilitating conditions.
Conclusions:
- Substantial research has not yet yielded an approved disease-modifying therapy for Parkinsonian disorders.
- Heterogeneity in disease mechanisms and limitations in clinical endpoints necessitate biomarker-driven and stratified trial designs.
- Future success hinges on improved patient selection, mechanistic targeting, and integrated biomarker strategies.
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