On January 7, Sarepta Therapeutics announced their topline results from their gene therapy candidate SRP-9001. This study is an ongoing, randomized, double-blinded, placebo-controlled trial evaluating the safety, efficacy and tolerability of a single dose of SRP-9001 in 41 Duchenne patients aged 4-7. The study met its biological primary endpoint of micro-dystrophin expression at 12 weeks. […]
How far would you go for someone you love?
That’s the question that led to the birth of Jesse’s Journey when one family dared to dream about what’s possible after learning the crushing news that Duchenne muscular dystrophy had become a part of their daily lives.
When John and Sherene Davidson learned that their middle son Jesse was diagnosed with Duchenne they charted a life changing course for themselves-setting out to raise research funds to find the elusive cure.