RESEARCH STRATEGY AND MISSION We aggressively pursue research to identify treatments and a cure for Rett syndrome. New Mecp2 female mouse model developed AMO receives FDA Orphan Drug Designation 2018 + BEYOND With your support, we can Neuren begins plans for trofinetide Phase 3 continue to blaze a trail in Rett syndrome research and family 2017 14 clinics designated empowerment to transform lives. as Rett Syndrome Clinical Research First multi-site, Centers of Excellence multi-country clinical Join us in our mission: trial begins: sarizotan • Make a donation 2015 • Coordinate a Fundraiser Clinical trial for • Participate in an Event trofinetide begins • Advocate for Rett syndrome NIH funding of the NHS begins Visit www.rettsyndrome.org or Drug screening Scout program begins call 1.800.719.8214 2014 First multi-site clinical trial Rettsyndrome.org is a 501(c)3 organization in RTT begins: NNZ-2566 (trofinetide) Accelerating dedicated to accelerating research for treatments and a cure for Rett syndrome and related disorders, 2013 while providing family empowerment. As a Established stem Research. leading organization for Rett syndrome research, cell model for Rettsyndrome.org is committed to funding high- drug screening First clinical trial quality, peer-reviewed research grants and programs. in RTT supported by Rettsyndrome.org: IGF-1 Empowering Genetic manipulation 2010 and biochemical Families. intervention improve Rett-like symptoms in a mouse model 2007 4600 Devitt Drive Cincinnati, OH 45246-1104 ‘‘ (800) 818-7388 www.rettsyndrome.org I am very thankful that Rettsyndrome.org has taken such a strong leadership role /rettsyndrome /rettsyndrome /rettsyndromeorg with advancing research. Their progress to get trofinetide to market is very exciting as it could finally be an answer to relieving some of Jill’s daily struggles. Rettsyndrome.org is the only organization committed to research and family empowerment. Imagine the symptoms WE BELIEVE • The cure for Rett syndrome depends on of autism, cerebral palsy integrating both treatments and gene and epilepsy affecting one therapies with physical, occupational and speech therapies child who cannot speak... • We have an obligation to support the Photo by this is Rett syndrome. families affected by Rett syndrome while Paul Vincent Kuntz Rettsyndrome.org is relentlessly pursuing treatments and a cure. committed to empowering WE HAVE RETT SYNDROME IS families. We offer resources, • Funded over $44 million in research RETT SYNDROME CLINICS AND NATURAL HISTORY STUDY A debilitating neurological disorder education, connections and • Invested over $10 million in family programs caused by a mutation of the MECP2 support so families can make and services Rett syndrome specialized care helps to improve a child's quality of life. Many Rett gene. It affects 1 in 10,000 female the best decisions for their • Developed our proprietary Scout Program syndrome clinics also participate in the to screen compounds for potential use in births. Rett syndrome is very rare in Natural History Study. child at any stage of life. Rett syndrome males. Children with Rett syndrome • The Natural History Study for Rett syndrome understand more than they with support from the NIH can communicate. • A clinical care network of 19 clinics that provide state of the art care to Rett syndrome patients Children often experience: • Breathing difficulties During this time of such tremendous RECENT HIGHLIGHTS • Cardiac issues progress in research, it is essential that • $2.4 million awarded for 2018 research projects. • Swallowing and ‘‘ we continue to provide services that • 3 compounds tested in the Scout program digestive issues in or planning for clinical trials • Scoliosis support the families affected by Rett Funding research into the early work of syndrome. We never forget that Rett • • Seizures gene therapy • Repetitive hand movements syndrome affects the entire family and For a full list of Rett Clinics and Natural History • Provide global access to Rett syndrome Study sites and contact information, visit our dedication to them is unwavering. experts to families through online webinars. Symptoms may be more severe in boys. www.rettsyndrome.org/natural-history-study Melissa Kennedy, Executive Director Rettsyndrome.org Girls and boys of any age are encouraged to participate in the Natural History Study!.