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Failure of Genetic Therapies for Huntington's Devastates Community


Scientific American
Hopes were high for drugs designed to lower levels of a mutant protein, but development has stalled
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Two pharmaceutical companies have halted clinical trials of gene-targeting therapies for Huntington’s disease (HD), following the drugs’ disappointing performance.
Researchers had hoped that the treatments known as antisense oligonucleotides (ASOs) would be a game changer for HD, an incurable genetic condition that affects cognition, behaviour and movement. But back-to-back announcements from Roche, headquartered in Basel, Switzerland, and Wave Life Sciences, in Cambridge, Massachusetts, have dealt a crushing blow to those affected by the disease.
“I was really shocked, really tearful,” says Marion, a woman in London with HD, who was part of one of the trials. “We didn’t see it coming at all. I felt really frightened and worried about my future.” Marion requested that her last name be withheld to protec ....

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