Sarepta Therapeutics has provided an update on the four boys participating in its gene therapy trial for Duchenne muscular dystrophy. It’s now been nine months since the boys were treated. They are all doing well and no serious safety concerns have been reported. Tests have also shown that their creatine kinase levels have decreased and their muscle function appears to be better than what is usually seen in Duchenne patients of the same age. Although the boys will continue to be followed up for 36 months in total, these results are promising and the company is now recruiting for a Phase II trial in USA.
For more information, access the conference call slides and audio here.