ALS Trial Shows Novel Therapy is Safe

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An article from highlights the exciting new results from Dr. Miller’s first Phase I trial of SOD1-targeting ASOs, stating that the investigational treatment for inherited forms of ALS has passed an early clinical trial for safety. “These results let us move forward in the development of this treatment and also suggest that it’s time to think about applying this same approach to other mutated genes that cause central nervous system disorders,” says lead author Timothy Miller, assistant professor of neurology at Washington University in St. Louis.