How big could a potential new drug for Huntington's disease be? The BBC puts it this way: "Experts say it could be the biggest breakthrough in neurodegenerative diseases for 50 years." The news is preliminary—full trial results should be published in 2018—but early indications are good. As the Guardian explains, the only currently available treatments address solely symptoms, allowing the disease to march on to its deadly conclusion, which can occur within a decade of diagnosis.
But this drug, called Ionis-HTTRx, appears to have slowed the progression. CNN reports Huntington's is caused by a single gene defect that causes cells to produce a toxic form of the huntingtin protein, which kills nerve cells in the brain. "You end up in almost a vegetative state," says a trial participant. Ionis-HTTRx prevents the toxic protein from being made.
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