A Roadmap to Cure Rett Syndrome

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I joined the Rett Syndrome Research Trust a year ago as Chief Scientific Officer with the specific goal of curing Rett Syndrome. This was not an aspirational goal, but a realistic one based upon a critical analysis of the research landscape. A series of breakthrough scientific discoveries have clearly demonstrated that Rett can be reversed in animal models and technologic advances are providing the tools to translate the science into human therapeutics.

Progress over the past year has exceeded my expectations, and I am proud to inform you that we just announced our three-year, $33 million transformational research plan to cure Rett Syndrome - Roadmap to a Cure.

At the core of the Roadmap are four parallel curative approaches, all attacking the root cause of the disorder, mutations in the MECP2 gene. Gene therapy, the delivery of healthy copies of the MECP2 gene to compensate for the mutated copy, is the most advanced and will be our lead program. Contingent on sufficient funding and requisite FDA approvals, the Roadmap is anticipated to enable the first clinical trial of gene therapy for people with Rett Syndrome.

In addition to my personal commitment to these efforts, Lynn and I encourage you to take advantage of our offer to double the impact of your contribution. We have committed to matching, dollar for dollar, the first $100,000 donated to our Rett Give portal. Our ultimate goal is to put RSRT out of business, and never again request another donation. You have my solemn promise, I will never ask you again for a donation to this or any other cause.

You’d be hard-pressed to find a more worthy cause than curing this devastating disorder. My first personal introduction was to a beautiful, strong, and resilient girl exactly the same age as my beautiful, strong and resilient daughter, 17 years old. Yet the contrast in hopes and dreams for our daughters was humbling. Specifically, because of a random spontaneous genetic mutation, these parents were not agonizing over SAT scores, college admissions or the appropriate curfew to set for prom night. Rather, they were faced with the likelihood that their daughter would never live independently, get married or have children. Seeing the struggles of every girl and woman with Rett drives me to do everything in my power to change their lives. We absolutely must succeed in our quest for a cure, and we can only succeed with your support.

You’d also be hard-pressed to find a more effective organization (96% of every dollar raised has funded research). Since inception in 2008, RSRT has provided over $41 million of research funding; funding that has enabled much of the breakthrough research that provides the foundation for our cure Roadmap. To date, virtually every penny has been donated by someone with a personal connection to an individual with Rett Syndrome. We now need to expand our donor base to enable a cure. I hope you will join us.


- Randy Carpenter


What is Rett Syndrome?

Rett Syndrome is serious lifelong neurological disorder that is caused by random mutations in a gene called MECP2. Diagnosed almost exclusively in girls, symptoms typically appear in toddlerhood. Many children with Rett are unable to speak, walk or use their hands. Breathing problems, feeding tubes, seizures, anxiety, gastrointestinal and orthopedic issues are common.

Despite the debilitating nature of the disorder our girls are so much more than a laundry list of symptoms. Their beauty and their strength inspire and motivate us.

Rett Syndrome stands apart from other neurological disorders due to the dramatic reversibility of symptoms in animal models. Our confidence that Rett Syndrome is curable guides our every decision.

Learn more about Rett here.

Who is RSRT?

The Rett Syndrome Research Trust (RSRT) was launched in 2008 with a singular goal: to drive the development of treatments and cures for Rett Syndrome and related MECP2 disorders. As one of the few neurological disorders to demonstrate dramatic symptom reversal in the lab, Rett Syndrome has captured the attention of the world’s premiere university and pharmaceutical industry scientists. RSRT is operating at the nexus of this activity, leading and advancing the research agenda.

By funding the most Rett research worldwide, $35 million since 2008, we strive to put ourselves out of business as soon as possible.

Learn more about RSRT here.

Supporters (68)

  1. Stephen Perna 
    1 year ago
    $250
  2. Tim & Cathy Babington 
    1 year ago
    $5,000
  3. John & Dorothy Remondi 
    1 year ago
    $1,000
  4. Paul Pospisil 
    1 year ago
    $100
  5. Owen McManus 
    1 year ago
    $500
  6. Thomas Moore 
    1 year ago
    $500
  7. Michael Walsh 
    1 year ago
    $100
  8. Joseph Horrigan 
    1 year ago
    $100
  9. Barbara Kelley 
    1 year ago
    $100
  10. Martha Childs 
    1 year ago
    $100
  11. Nancy Satwicz 
    1 year ago
    $500
  12. Hani Yaqo 
    1 year ago
    $100
  13. Craig Chisholm 
    1 year ago
    $30
  14. Steven Barrett 
    1 year ago
    $100
  15. David Lanzetta 
    1 year ago
    $100
  16. Jerry Kroft 
    1 year ago
    $100
  17. steven carpenter 
    1 year ago
    $50
  18. Eric Huang 
    1 year ago
    $500
  19. Michael Greenberg 
    1 year ago
    $250
  20. Kathy Roberts 
    1 year ago
    $1,000
  21. Deborah Dunsire 
    1 year ago
    $500
  22. Jeff Davis 
    1 year ago
    $100
  23. Lisa Paborsky 
    1 year ago
    $250
  24. Geoffrey Hardie 
    1 year ago
    $100
  25. randal bourjaily 
    1 year ago
    $500
  26. Donald Volkmann 
    1 year ago
    $100
  27. Sean and Emily Babington 
    1 year ago
    $100
  28. Tim & Cathy Babington 
    1 year ago
    $2,500
  29. Linda Malone 
    1 year ago
    $500
  30. Paul and Annette LaCasse 
    1 year ago
    $1,000
  31. Brian Harrington 
    1 year ago
    $500
  32. Nick Galakatos 
    1 year ago
    $2,500
  33. Nabil Uddin 
    1 year ago
    $100
  34. AILEEN HEALY 
    1 year ago
    $250
  35. Caitlyn Carpenter 
    1 year ago
    $100
  36. Mark Levin 
    1 year ago
    $1,000
  37. Jeroen Verheijen 
    1 year ago
    $50
  38. Thomas Hubbard 
    1 year ago
    $100
  39. Adelbert Spitzer 
    1 year ago
    $500
  40. Amy and Geoff Somary 
    1 year ago
    $1,000
  41. David Margulies 
    1 year ago
    $500
  42. Philippe Amouyal 
    1 year ago
    $2,500
  43. Melincoff Family Charitable Fund 
    1 year ago
    $500
  44. Christian Carpenter 
    1 year ago
    $100
  45. Henry Goodrow 
    1 year ago
    $1,000
  46. Jack Morgan 
    1 year ago
    $150
  47. TImothy Ocain 
    1 year ago
    $1,000
  48. Michael Clayman 
    1 year ago
    $250
  49. Russell and Sooni Carpenter 
    1 year ago
    $100
  50. DAVID JAYE 
    1 year ago
    $1,000
  51. Joseph Gonzalez-Heydrich 
    1 year ago
    $100
  52. William Pentelovitch 
    1 year ago
    $1,000
  53. Margaret Phillips 
    1 year ago
    $50
  54. Stephen Rupp 
    1 year ago
    $100
  55. Anonymous 
    1 year ago
    $100
  56. Michael Palfreyman 
    1 year ago
    $100
  57. Steven Carpenter 
    1 year ago
    $100
  58. David Randall 
    1 year ago
    $100
  59. randi hagerman 
    1 year ago
    $100
  60. Leonard Rappaport 
    1 year ago
    $100
  61. Peter Zarevics 
    1 year ago
    $500
  62. Jason Rhodes 
    1 year ago
    $500
  63. William Schmidt 
    1 year ago
    $500
  64. Joseph Faber 
    1 year ago
    $1,000
  65. Lynn Babington & Randy Carpenter 
    1 year ago
    $46,950
  66. Mark and Cindy Dowd Greene 
    1 year ago
    $200
  67. Errik Anderson 
    1 year ago
    $100
  68. Bruce Tofias 
    1 year ago
    $100

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