The Path Forward

There is a program here that has the potential to stop the progression of the disease in more advanced subjects and actually cure the disease in newly diagnosed. Wouldn’t it be nice IF another family never had to hold their child in their arms and watch them fade away………….IF another family never had to live every day wondering IF today is the last day for their child. Imagine IF we as parents never had to watch our kids progress in this disease, IF we did not have to watch as our child lost arm movement………….or watch as they struggle to finish what we know will be their last bottle…………IF we did not have to watch as the open and closing of their hands is reduced to merely moving a finger. Imagine IF we did not have to witness the degeneration as a beautiful smile disappears……………..Right now we can only imagine IF.

There is no therapy that comes with a guarantee that it will translate to humans………..However there has never been a program with better Pre-clinical data. Never has there been a program with a combination of such promising safety AND efficacy. ┬áThe long term spread out approach of funding is thoroughly covered by other groups. We are focused on bringing a possible treatment to this community NOW!

We cannot fund this alone……….we need YOU to help turn this into a reality. The AAV9 Gene Therapy program is in dire need of $1,000,000 in funding and right now there are no commitments to see this funded. We cannot turn our backs on these kids now. Similar programs have received $2,000,000 to $15,000,000 in funding from the community. To date the AAV9 at Nationwide has seen a combined total of $600,000 and we need to raise $1,000,000 more to move this into clinical trials in a year. That timeline and these required studies are the guidelines that have been set forth by a Pre Pre Ind conversation with the FDA.

This is going to require a monumental effort by all of us to see this through! We cannot wait to see who may help in the future………and $1,000,000 in a year will require action now. This can become a reality but we must work together. We need 200 commitments of $5,000. Are you willing to commit to raising $5,000 by March 9, 2012? If you can raise $10,000 that is great ………that would take care of 2 commitments. In order to move forward with this program we need to know who is on board. Whether you are an individual, a family, a non-profit, a company you can get involved. Are you willing to take a $5,000 chance on this program?

Please email if you are interested in helping!

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