Advancing ASO therapies from development to implementation
Nature Medicine, Published online: 13 September 2024; doi:10.1038/s41591-024-03217-x
A novel application of antisense oligonucleotide (ASO) technology, developed to treat a single patient, adds to the growing number of ‘personalized’ therapies for rare diseases; but pathways to implementation and access are urgently needed.
Advancing ASO therapies from development to implementation
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