Vision and Guts

Our recent announcement that AveXis is planning to initiate a gene therapy clinical trial within a year (pending FDA approval) is exciting and worthy of celebration. Getting us to this point took years of hard work and millions of dollars raised and thoughtfully awarded. It is truly a moment worthy of stepping back and recognizing the effort of so many people – our scientists, our families who fundraise, our generous donors, our relentless trustees and our passionate staff. We would not be at this point without you.

Nevertheless, this is not the time to think that our work is done – that we can coast from here on out. In actuality this is the time to double down. Here’s why:

1. While getting to the trial is a big step, it’s the results that matter. And we don’t know what those results will be. Will they be dramatic or not? Will all symptoms be addressed or not? Will older children and adults benefit? Until we have a cure or dramatic improvements in hand, our work must continue.

2. We have a solid research plan to follow, Roadmap to a Cure, that prioritizes not only gene therapy but also three other approaches that attack Rett Syndrome at its genetic core. Each of these approaches has to be aggressively pursued until we know for certain where we will find a cure.

3. We have the staff, scientists and advisors to execute on this bold plan. And importantly we have the vision and the guts to act boldly. Pursuing strategies like gene therapy is not for the faint of heart.

4. Our business model is to invest significant dollars in high impact areas such as gene therapy via collaborative Consortia who generate sufficient data to attract an industry partner. We now have proof that this strategy works and we will apply that model to other areas of our research portfolio.

I hope the AveXis news has inspired you. If you are actively fundraising for us, I am beyond grateful. Please continue to do so with renewed optimism and determination.

If you are not fundraising for us I ask that you consider channeling that inspiration into action. We are in the midst of a groundswell movement with families in the US and around the world taking charge of their children’s future. I invite you to be a part of that groundswell!

Contact us to get started.