Rett Syndrome Awareness Month has been incredible. As a community, you rallied like never before – speaking out to share the good, the bad, and the ugly about Rett syndrome to educate the world and move the needle. Your efforts continue to make a difference. Today, more people know about Rett syndrome than ever before – neighbors, communities, officials, donors, and researchers! Raising awareness fuels our rare disease research effort and has allowed us to invest millions in research to date but, more importantly, has driven investments from our partners in biotech, pharma, and the U.S. Government. Rettsyndrome.org sponsored Legislative Days have facilitated parents and professionals speaking to Congress on behalf of the Rett syndrome community, resulting in tens of millions of dollars being allocated in the budgets of the National Institutes of Health and the Department of Defense, specifically for Rett syndrome research. These ‘partner’ investments in Rett research have catalyzed large-scale research projects that could not be financed by a private foundation support alone.
COLLABORATION WITHIN RETT RESEARCH
Just this week, Rettsyndrome.org had the privilege to co-host a once-in-a-lifetime research symposium in Houston with RSRT, chaired by Dr. Huda Zoghbi and Sir Adrian Bird. Nearly 300 Rett syndrome investigators, researchers, clinicians, members of the NINDS, pharmaceutical companies, and experts from around the world came together to discuss the future direction of Rett research; twenty years ago, this would not have been impossible! The ideas and suggestions arising from the symposium will significantly influence Rettsyndrome.org’s research strategy because we believe it is our responsibility to focus on research that brings the best treatment options and ultimately reach toward a cure.
CLINICAL TRIAL DEVELOPMENTS
And on the heels of the research symposium, ACADIA Pharmaceuticals announced the launch of their Phase 3 clinical trial for Trofinetide, a compound that without Rettsyndrome.org support, would not be where it is today. Three sites are screening now, and thirteen sites will begin actively screening soon.
Our commitment to fuel research with results remains unwavering. Today we have supported the investigation of 4 compounds currently in clinical trials. We are also regularly advising pharmaceutical companies to help them design clinical trials that are best suited for our community, regardless of whether we funded the compound being considered. Our full-spectrum research approach includes a focus on neuro-habilitation, the physical, occupational, speech, and cognitive therapies needed to regain the skills and abilities Rett has stolen. This essential work needs to be done in tandem with drug therapies and is an important element in the day to day life of an individual impacted by Rett syndrome.
RESEARCH AND FAMILY EMPOWERMENT
We believe a foundation can advance research while empowering families; we’ve been doing it for 20 years. In addition to our proven research strategy, we are proud to offer support, education, and countless resources to empower families. We have four individuals on staff and five members of our Board of Directors who have children with Rett syndrome. We understand the need for hope; our support and resources offer just that.
A newly diagnosed family recently shared during Rett Syndrome Awareness Month, “When Lexi was first diagnosed, the Rettsyndrome.org’s family empowerment team provided our family great resources, connected us to other local families, and provided overall support. In the chaotic, overwhelming days when we first received the diagnosis, this amazing organization provided much-needed support as we navigated the way ahead for Lexi’s care. Part of the money raised for Rettsyndrome.org not only goes to medical research for a cure but also to resources for newly diagnosed families like ours.”
Here are a few more ways we have supported families this year:
- Family and Community Engagement Manager (and Rett mom), Samantha Brant, offers counsel and support to families affected by Rett syndrome.
- Our volunteer Family Empowerment representatives walk with families and connect them to local resources and families.
- Our educational resources are unmatched.
- RSO hosted 3 Ed Days across the U.S. and partnered with other Rett associations and clinics to enhance their Educational Days.
- RSO hosts monthly RettEd Webinars featuring leading experts in research and topics relevant to families.
- Over 1,600 individuals from 54 countries participated in our educational programs.
- Our educational programming is underwritten by grants from industry partners
- We launched the Rett Syndrome Primary Care Guidelines – the first of its kind!
- Our new website offers countless resources for free! And we continually update those resources so that you can easily find information, clinics, clinical trials, and expert advice.
Your families keep us focused on both research and empowerment. We are honored to serve this community of passionate, resilient, and hard-working families, caretakers, researchers, and advocates. Together, we have accomplished great things, and the greatest is yet to come!
We remain unwavering in our mission to serve your family while focusing on creating a world without Rett syndrome.
The Rettsyndrome.org Team