STAT+: Alnylam says Huntington’s drug shows promise in early study

·STAT News··

An experimental Huntington’s drug from Alnylam Pharmaceuticals showed promise in a small study, the company said Friday, suggesting it can partially silence the mutant gene that drives the devastating neurological disorder. Patients who received the highest dose of the drug, called brezosiran, saw the levels of mutant huntingtin in their spinal fluid fall by 60% or 64% on average, depending on the type of assay used. The company described the safety profile as “encouraging” and said it planned t...

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