When we incorporated Charley’s Fund in November 2004, I had no idea that we could have an impact on the medical research in such a short period of time. Mentally, I buckled in for a very long road, expecting to raise money and then wait for months – or even years – to see if it helped. So I am amazed to report that in just 18 months, we have already seen tangible results on several fronts.
Our most exciting development: our research team in the Netherlands has started a human clinical trial of their exon skipping therapy. Exon skipping modifies the way the body reads the genetic code. Boys with Duchenne who are successfully treated with exon skipping will “convert