New top-line data from a Phase I/II study of Ionis Pharmaceuticals Inc.'s investigational Huntington's disease antisense product IONIS-HTTRx (RG6042) suggest it can significantly reduce levels of the disease-causing mutant huntingtin protein, making it the first product to show disease-modifying potential in the disease.
Partner Roche is now speeding the product, which has US and EU orphan drug status, into a pivotal study.
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