This week, at the 23rd International Congress of the World Muscle Society in Argentina, Dr. Jerry Mendell of Nationwide Children’s Hospital in Columbus, Ohio, shared additional data relating to Sarepta Therapeutics’ AAVrh74.MHCK7.micro-Dystrophin gene therapy program for Duchenne muscular dystrophy (DMD). 

Dr. Mendell had previously presented results for the first three patients in the study back in June. At this week’s meeting, he shared micro-dystrophin results for the fourth patient and reported positive functional results for all four patients in the trial. These patients showed improvement in several metrics, including time to rise, time to climb four stairs and time to walk 100 meters. Additionally, all four patients showed robust expression of the transduced micro-dystrophin gene. No serious adverse effects were observed.

It is important to remember that while these results are encouraging, they need to be confirmed by a controlled trial.

To learn more, visit Sarepta’s website.