Action Duchenne and Duchenne Ireland funds further research for exon skipping therapies | Response Source

Action Duchenne and Duchenne Ireland funds further research for exon skipping therapies |
The new research will investigate the possibility of ‘multi’ exon skipping, thus enabling more exons to be targeted with the treatment of AOs (not just exon 51) and benefiting larger groups of patients.

According to Dr. Matthew Wood; “Although more than 80% of Duchenne boys could potentially benefit from the “exon skipping” approach, each exon targeted requires a unique AO, and thus treating all the different mutations known to cause DMD requires the development of large numbers of AOs, each treating only a small subset of the patient population.