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Antisense oligonucleotides in neurological disorders.

Ther Adv Neurol Disord · 2018 · PMC5971383 · PMID 29854003

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However, the encouraging clinical effects under the continuous treatment with drisapersen compared with placebo did not reach statistical significance by week 49. 86 , 87 A subsequent and larger double-blind phase III study investigated 186 patients randomized to drisapersen (6 mg/kg/week) or to placebo for 48 weeks.

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