Exon skipping is a potential treatment approach for correcting and restoring production of dystrophin.
For specific genetic mutations, it allows the body to make a shorter,
usable dystrophin. Exon skipping is not a cure for Duchenne, but it
may make the effects less severe. No single drug will help everyone
with Duchenne. The most common mutation amenable to exon skipping is
exon 51, which only affects 13% of all patients. But doctors believe
that 60-80% of Duchenne patients may eventually benefit from exon
Duchenne Population Amenable to Exon Skipping
Data suggest up to 80% of patients have genotypes amenable to exon skipping.