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Publications

Over 200 publications, including the composite endpoints regulators now recognize. We publish our methodology in the open so reviewers meet it before your submission does.

200+
Publications
1996–2026
Span
28
Contributing authors
White paper

Global Statistical Tests: powering the trial your budget can actually fund

When the sample size a conventional design demands is larger than the trial you can run, a Global Statistical Test lets the clinical-endpoint hypotheses be tested anyway. The paper sets out when GST applies, how it is pre-specified, and how it has been received in review.

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Research

Publications library

1 publication

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  1. 2016 · Immunotherapy and Biomarkers in Neurodegenerative Disorders

    Immunotherapy of Parkinson's Disease

    Hendrix, Suzanne

    Abstract

    Abstract: Parkinson’s disease (PD) is the second most common neurodegenerative disorder. It elicits a broad range of debilitating motor and as well as non-motor symptoms, both of which can lead to serious disability. There is currently no available agent with disease modifying properties. Immunotherapy is increasingly being investigated as a disease modifying treatment for PD based on our improved understanding of the pathophysiology of the disease. Current evidence points to a causal role of misfolded alpha-synuclein (α-syn) in the development and progression of PD and it has therefore become a primary focus for immunotherapy. Today, two principal approaches are being pursued: active and passive immunization. This chapter first addresses progress in active and passive immunotherapeutic approaches targeting α-syn for Parkinson’s disease in animal models. We then discuss clinical progress of α-syn immunotherapy including ongoing clinical trials. Finally, we address challenges and future perspectives for PD immunotherapy.

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