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Ionis Announces Positive Top-Line Data

From a completed Phase 1/2 study of IONIS-HTTRx (RG6042) in people with early stage Huntington's disease

Ionis Pharmaceuticals has presented positive top-line data from a completed Phase 1/2 study of IONIS-HTTRx (RG6042) in people with early stage Huntington’s disease. The data demonstrates that IONIS-HTTRx (RG6042) is the first drug in development to lower the disease-causing protein in people with HD.    HD is a rare, progressive, neurodegenerative disease caused by genetic mutation in the huntingtin gene, which results in the production of a toxic protein, the mutant huntingtin (mH...

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