We are beyond thrilled to announce that three Duchenne families from across the country have committed half a million dollars to match what CureDuchenne raises in our year-end fundraising efforts!
These families are partnering with CureDuchenne to help further our 2019 funding projects into research that will ensure effective drugs are available to all Duchenne boys and young men — regardless of the progress of their disease.
Today, there is hope for our boys. This year alone, we witnessed substantial gains in our mission to cure Duchenne, with published research by two of our partners. Dr. Eric Olson and his team at Exonics, a CRISPR/Cas9 gene editing company that CureDuchenne helped form, published data showing how a genetic correction of the dystrophin gene led to an increase in dystrophin by 92% in the heart of canines with Duchenne. Sarepta Therapeutics, another biotech partner we contributed to very early, published news of up to 96% increase in mini-dystrophin in four Duchenne boys dosed in its gene therapy clinical trial.
We now have a clear roadmap for how to cure Duchenne — we are in the final home stretch. With a goal to raise $1 million by December 31st, we kindly ask once again for your support to help us meet this objective and accelerate the science to save this generation of boys and young men.
We are dedicated to supporting next generation therapies that will dramatically increase the effectiveness — and truly lead to a cure. And with your help, we’ll continue making strides in Duchenne care, keeping our boys out of wheelchairs and giving them the lives they were intended to live – to graduate from college, to contribute to their communities, to fall in love and to have children of their own.
Debra and Paul Miller