NewsJOURNAL OF NEUROMUSCULAR DISEASESThursday, March 5, 2026 · March 5, 2026
Antisense RNA therapies for muscular dystrophies.
WHY IT MATTERS
Recent peer-reviewed research on Duchenne muscular dystrophy that may be relevant for patients and caregivers.
Inherited muscular dystrophies are a heterogeneous group of diseases, caused by different types of genetic mutations. RNA therapies, and particularly antisense oligonucleotides, offer a palette of therapeutic strategies to either reduce the production of harmful proteins or to restore or increase pr...
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