1. Early Phase Disease Modification Trials with Selisistat and Optical Coherence Tomography as a Biomarker in Huntington’s Disease (Thesis)

    Early Phase Disease Modification Trials with Selisistat and Optical Coherence Tomography as a Biomarker in Huntington’s Disease (Thesis)

    Huntington’s disease (HD) is a monogenic neurodegenerative disorder with no known cure. Selisistat is a novel, highly potent Sirt1 inhibitor with supportive pre-clinical data. Facilitation of autophagy and amelioration of transcriptional dysregulation are proposed as mechanisms of action. A first in disease Phase 1B study showed safety and tolerability. The peripheral immune dysfunction in HD could be modulated by Sirtuins which have both pro and anti-inflammatory activities. However selisistat did not alter the cytokine profile in this study. Phase 2 data over a longer duration was also well tolerated however potential hepatotoxicity is a concern. Sub-analysis of clinical assessments ...

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