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Rett Superheroes

January 4, 2019

We launched Roadmap to a Cure, RSRT’s three-year $33 million strategic research plan, in March of 2017. We’re now 21 months into the 36-month period, 60% through the timeframe. I’m excited to report that as of January 1 we’ve raised almost $24 million towards the goal. That’s 73% of the way there. We’re ahead of schedule!

Nothing I can say here adequately expresses the gratitude I feel to every one of RSRT’s supporters. I know my RSRT colleagues, our trustees, and every family affected by Rett feel the same gratitude that I do. To the families that fundraise for us, my thanks and admiration is deeply felt and personal. Whether you are one of our Roadmap Trailblazers or you fundraise for us through an event or RettGive or Facebook, you should be very proud. We would not be here without you.

$24 million is such a big number that it’s hard to visualize, so I did a little googling to help me wrap my own mind around it. I learned that if you stacked up a million one-dollar bills it would measure 358 feet, about the height of a 30-story building. If you stacked up 24 million one-dollar bills it would stretch more than a mile-and-a-half, way taller than any building. The $33 million we need to raise would be over 2.2 miles high if stacked in bills!

That gave me a fun visual on what all who are involved with RSRT have built with your generosity and efforts. Far more important is what those dollars have really made possible. The first human clinical trial in gene therapy is on track to begin this year; a second-generation gene therapy is being developed to ensure maximum impact on lives; potentially curative approaches in MECP2 reactivation, RNA editing and transsplicing, and protein replacement are making promising strides; five leading clinics are members of the Clinical Trial Consortium and are setting the stage for the highest quality clinical trials. Spearheading all this are Monica Coenraads, Randy Carpenter, Tim Riley, and Jana von Hehn, who never cease to amaze me with their knowledge and dedication and their ability to recruit and empower world-class researchers to attack Rett Syndrome with the boldest approaches that are at the vanguard of science. In short, I have never been more optimistic about the future, and I mean the near future, for all with Rett, including my own daughter. The $24 million raised so far has made all this possible.

We have $9 million more to raise to fully carry out the priority approaches of Roadmap to a Cure. That’s going to take the continued energy of families that are already fundraising, and it’s going to take more families getting involved with us. As far as I’m concerned, all who helped get us this far are superheroes, capable of leaping tall buildings, including ones that are a mile-and-a-half tall! If you’re not involved with us yet, join the superheroes who already are and help us take the next leap over the 2.2-mile-high building. We need you more than ever. Are you in?