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Antisense oligonucleotide skips exon 53 in DMD patients

By John Fox
Staff Writer

Tuesday, April 24, 2018

HONG KONG – An antisense oligonucleotide (ASO) that induces skipping of exon 53 has been shown to have a favorable safety profile and promising pharmacokinetics in children with Duchenne muscular dystrophy (DMD) in a Japanese phase I trial. That warrants further study of the ASO in a phase II study, researchers led by Shin'ichi Takeda, director of the Department of Molecular Therapy National Institute of Neuroscience, National Center of Neurology and Psychiatry (NCNP) in Tokyo, reported April 18, 2018, in Science Translational Medicine.

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