Alnylam's Huntington's drug brezosiran cuts mutant huntingtin in early study
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Alnylam has reported results from a small early study of brezosiran, its experimental Huntington's disease drug. In the highest-dose group, mutant huntingtin levels in cerebrospinal fluid fell by 60% or 64% on average. The company said the drug showed good safety and tolerability. The data were presented at the European Huntington's Disease Network Clinical Research Congress. Alnylam plans to start a placebo-controlled study next year to test whether brezosiran can slow disease progression. That outcome study, not the biomarker reduction, will determine the drug's real value; the current results come from a small, uncontrolled trial.
Written by a model from the reports · updated 2 hours ago
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- statnews.comPickSTAT+: Alnylam says Huntington’s drug shows promise in early study
Alnylam reported results from a small early study of brezosiran, in which the highest dose reduced mutant huntingtin levels in cerebrospinal fluid by an average of 60% or 64%. The company described the drug as having good safety and tolerability and plans to start a placebo-controlled study next year to test whether it can slow disease progression. The data were presented at the European Huntington's Disease Network Clinical Research Congress.
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