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IONIS-HTT Rx (RG6042) Top-Line Data Demonstrate Significant Reductions of Disease-Causing Mutant Huntingtin Protein in People with Huntington’s Disease

Phase 1/2 study first to demonstrate disease-modifying potential Up to ~60% reduction (mean ~40%) in the mutant huntingtin protein (mHTT) observed in the cerebral spinal fluid (CSF) of patients treated for three months with IONIS-HTT Rx (RG6042) at the two highest doses tested with mHTT levels