Newborn screening of duchenne muscular dystrophy specifically targeting deletions amenable to exon-skipping therapy
Abstract Duchenne Muscular Dystrophy (DMD) is a lethal progressive muscle-wasting disease. New treatment strategies relying on DMD gene exon-skipping therapy have recently been approved and about 30% of patients could be amenable to exon 51, 53 or 45 skipping. We evaluated the spectrum of deletions...
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| Hlavní autoři: | , , , , , , , |
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| Médium: | Artigo |
| Jazyk: | Inglês |
| Vydáno: |
Nature Portfolio
2021-02-01
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| Edice: | Scientific Reports |
| On-line přístup: | https://doi.org/10.1038/s41598-021-82725-z |
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