Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy
<p><strong>Objective:</strong> To report safety, pharmacokinetics, exon 53 skipping, and dystrophin expression in golodirsen-treated patients with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping.</p> <p><strong>Methods:</strong> Part 1 was a...
| Main Authors: | , , , , , , , , , , , , , , |
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| Format: | Journal article |
| Language: | English |
| Published: |
American Academy of Neurology
2020
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