Developing DMD therapeutics: a review of the effectiveness of small molecules, stop-codon readthrough, dystrophin gene replacement, and exon-skipping therapies.
Expert Opin Investig Drugs. 2021 Feb;30(2):167-176. doi: 10.1080/13543784.2021.1868434. Epub 2021 Jan 6.
Expert Opin Investig Drugs. 2021.
PMID: 33393390
Review.