Viltolarsen in Japanese Duchenne muscular dystrophy patients: A phase 1/2 study.

Ann Clin Transl Neurol · 2020 · PMC7732240 · PMID 33285037

Europe PMC DOI

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an increasing trendno p-value reported

Interpretation Treatment with viltolarsen 40 or 80 mg/kg elicited an increasing trend in dystrophin expression and exon 53 skipping levels, and was safe and well tolerated.

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