By Jocelyn Duff

It is hard to believe that we have reached this first milestone after having only been at it for a little over a month. We have achieved so much in such little time. We are so incredibly grateful to everyone who has contributed to The Talia Duff Foundation to Cure CMT4J. From bake sales to lemonade stands, to fishing derbies, to the wave of support for our YouCaring page, the love and support from our community, family, friends and strangers is overwhelming.

Our team is is also growing quickly. Somehow we were smart enough to know that we would need a lot of help to get to our goal of developing a treatment or cure for CMT4J. Our Board of Directors boasts a leader in development for a major Boston research hospital; a Senior executive in strategy and corporate development and a creator of one of the internet’s first online news operations. We have some of the smartest, most creative people on our broader team who are helping us with business plans, non-profit operations, reaching out to the rare disease community, bridging with the medical and scientific community, working on development and fundraising, and keeping us on course to look at all possible ways that we can get to our ultimate goal as fast as possible.

Our scientific team is slated to meet early this Fall. Led by Dr. Jun Li of Vanderbilt University, we have comprised a team of scientific investigators to address an expedited path toward a treatment or cure for CMT4J. We have leaders in gene therapy and viral vector development, scientists who know the mutation involved with CMT4J better than anyone, and physicians who have traveled the path of gene therapy from drug development to clinical trials.

Phew! We have a lot to celebrate! We also have a long way to go. As we enter Phase II, we look to our meeting of scientific investigators to guide us through. If, indeed, gene therapy holds the most promise, then we begin development of a viral vector and pre-clinical trials to test proof of concept. Our meeting will also give us a better sense of the resources needed for this phase. Generating awareness and fundraising will remain a priority for The Talia Duff Foundation and we appreciate the word-of-mouth and social media you have already helped to generate for our cause. Please keep the drumbeat going for us!

On those days when this task seems insurmountable, I remind myself that just a few months ago we had no hope. We did not know that a treatment or cure was possible. I hold my girl and I think about how different her life might be if we can get to the goal.