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

1 Dec 2020

Abstract excerpt

OBJECTIVE: The novel morpholino antisense oligonucleotide viltolarsen targets exon 53 of the dystrophin gene, and could be an effective treatment for patients with Duchenne muscular dystrophy (DMD). We investigated viltolarsen's ability to induce dystrophin expression and examined its safety in DMD patients. METHODS: In this open-label, multicenter, parallel-group, phase 1/2, exploratory study, 16 ambulant and...

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PMID: 33285037
DOI: 10.1002/acn3.51235

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