Abstract
Huntington's Disease: The Most Curable Incurable Brain Disorder?
Highlights
The more prevalent neurodegenerative diseases like Alzheimer's and Parkinson's lack this certainty: only a tiny minority of cases have a defined genetic cause
Large cohorts of patients and mutation carriers were assembled and studied over years, to determine what measurements were most reliable for predicting onset and progression
We reported the first quantification of mutant huntingtin protein in cerebrospinal fluid (CSF), and showed that its concentration predicts clinical features of HD
Summary
The more prevalent neurodegenerative diseases like Alzheimer's and Parkinson's lack this certainty: only a tiny minority of cases have a defined genetic cause. A failed drug does not imply a failed trial. These experiences improved the field's ability to design and run trials that can give a definitive answer, and to prioritise therapeutic targets and generate comprehensive preclinical data to inform decisions about advancing to clinical trials.
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