Clinical trials using antisense oligonucleotides in duchenne muscular dystrophy.
Duchenne muscular dystrophy (DMD) is a severe muscle wasting disorder caused by mutations in the DMD gene, affecting 1 in 3500 newborn males. Complete loss of muscle dystrophin protein causes progressive muscle weakness and heart and respiratory failure, leading to premature death. Antisense oligonu...
| Main Authors: | , |
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| Format: | Journal article |
| Language: | English |
| Published: |
2013
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