Exon-skipping therapy: a roadblock, detour, or bump in the road?
Where this comes from
- Record sourced from PubMed, PMID 24695683.
- Also identified by DOI 10.1126/scitranslmed.3008873 and PMC identifier 4464785.
- No licence information is recorded for this record.
- Because redistribution is not established, this page shows the abstract only. Follow the links below for the full text.
Abstract
Exon skipping is a promising therapeutic for Duchenne muscular dystrophy patients, but the road to drug approvals is foggy and may require more early-stage derisking and regulatory guidance.
Medical subject headings
- Exons
- Genetic Therapy