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Description
Dihexa is dosed individually as part of a supervised protocol

911 This extremely large structure means that all the multiple strategies employed in development of curative genetic approaches mini genes, gene editing, exon skipping, and stem-cell based treatments face significant logistic challenges

[DOI] [PMC free article] [PubMed] [Google Scholar] 624.Louvrou V., Solianik R., Brazaitis M., Erhardt S

Development 2006
