2 SMN2-targeting Therapies Work Better Than 1 in Mouse Model

2 SMN2-targeting Therapies Work Better Than 1 in Mouse Model

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2 SMN2-targeting Therapies Work Better Than 1 in Mouse Model

Treatment with two distinct molecules targeting the “backup” SMN2 gene resulted in higher levels of SMN — the protein lacking in spinal muscular atrophy (SMA) — and longer survival in a mouse model of type 1 disease relative to the use of a single therapy. Notably, these benefits, which included the prevention of motor neuron loss, were observed when the combination treatment was given both before and after symptom onset. The combination also was found to be generally…

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