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T. Simuni

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Review Open access Oct 2026

Considerations for therapeutic targets for clinical trials in biomarker-characterized prodromal Parkinson's disease populations

Parkinson's disease (PD) is a growing public health problem and biologically targeted therapeutics (BTTs) that slow disease progression are urgently needed. However, the development of effective BTTs is challenging as PD is a clinically defined syndrome with pathobiological heterogeneity and the pathobiological process...

R. Schneider, G. Skibinski, B. Fiske et al. · 0 citations
Review Open access Sep 2026

Progress towards a biotypic biomarker profile for amyotrophic lateral sclerosis-frontotemporal spectrum disorders.

An international workshop was convened in London, Canada in 2025 to review the evidence for existing or evolving biomarkers suitable for the detection of either ALS or FTD pathobiology prior to phenoconversion and/or predict phenoconversion in at risk individuals.

Michael J. Strong, Neil S. Donison, A. Al-Chalabi et al. · 0 citations
Review Open access Sep 2026

Cognition endpoints for Parkinson's disease: A roadmap for developing patient-focused assessment tools to accelerate drug development

Recommendations are made regarding necessary literature reviews, including qualitative data and related conceptual models, disease models including pathophysiology and neuropsychological profile, and clinical outcome assessment (COA) reviews, including content validity, psychometrics, and fitness-for-purpose.

Chris J. Edgar, Kathleen L. Poston, Cheryl D. Coon et al. · 0 citations
Review Aug 2026

TDP-43-Associated Neurodegenerative Disease Conceptualization and Integrated Staging: A Review.

A pathobiological definition of disease is proposed to provide a road map for developing biomarkers that accurately reflect the underlying pathobiology of disease and for advancing therapeutic candidates that effectively target fundamental disease mechanisms.

M. Benatar, SJ Barmada, Gregory A. Jicha et al. · 1 citation
Aug 2026

GCH1 genetic variation as a prognostic factor in Parkinson disease across populations

GCH1 pathogenic variants were associated with a clinically distinct phenotype characterized by earlier disease onset and slower progression of motor complications, suggesting that GCH1 genetic variants may serve as genetic biomarkers for patient stratification and prognosis in PD.

J. Shin, M. T. Periñán, J. W. Jang et al. · 0 citations

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