Golodirsen: First Approval.
Heo YA.
Drugs. 2020 Feb 6. doi: 10.1007/s40265-020-01267-2. [Epub ahead of print] Review.
https://dx.doi.org/10.1007/s40265-020-01267-2
This is an FDA-approved Morpholino drug that causes skipping of exon 53 of human dystrophin, used for the treatment of Duchenne muscular dystrophy.
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