In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse model.

With the 12-month data from the open-label extension of Tofersen, signs of reduced disease progression across multiple secondary and exploratory endpoints (including motor function, respiratory function and quality of life) were also observed, though the primary endpoint as measured by the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale did not reach statistical significance [ 14 ].

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