A Cure for Kate

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Today we are celebrating our daughter Kate’s 17th birthday.  She’s excited to be going with her friends bowling.  Sure, there will be some bumpers and some devices to help Kate and her friends get the ball down the alleyway, but what matters is that they are together and having fun!  She’s able to bowl now, but that might not always be the case.

For those of you who know Kate, you’re aware that she has suffered from developmental delays since she was young.  She struggles to speak clearly, her fine motor skills are almost non-existent, and she walks with a limp and struggles with balance.  Still, she faces each morning with a smile on her face and true joy in her heart.

Last year, Kate was diagnosed with Rett Syndrome through genetic analysis.  Rett is a developmental disorder seen almost always with girls.  Symptoms for girls with Rett include scoliosis, seizures, wringing of hands, bowel problems, and an inability to walk and talk. The specific mutation Kate has is rare even for Rett, which may be why she hasn’t suffered from all of the symptoms.

Now is an exciting time in research for Rett Syndrome.  Cell and gene therapies are showing real promise with certain rare genetic diseases.  There are no approved gene therapies in the US, but that’s about to change as there are three products in gene therapy applying for FDA approval in 2017.  These are not for Rett, but for other genetic diseases, but it shows what concerted research and clinical trials can do in less than a decade.   Best of all, these therapies can be limited to one or just a few doses over a lifetime!  It’s truly amazing.  We should know – Dave has been involved in gene therapy for over 20 years and it has really taken hold only over the past 6-7 years.

And now - the first clinical trial for Rett Syndrome using a gene therapy technique appears likely.  The people running the trials and funding the trials clearly have the desire to correct this devastating disease.  It may not work -  that is what the trials are for.  But we’ve never gotten this far and it’s not time to slow down.

So we ask you to support Kate on her birthday and throughout this year. We ask that you make a donation to help Kate retain her ability to walk and talk. Donations will be used for clinical trials that could directly help Kate and the other Rett girls.  We’ve selected support levels named after things that make Kate smile. Thank you from all of us for your continued love, support, and generosity.

Dave & Mary,

Grace, Kate & Claire Backer


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 (97)

  1. Patricia & Robert Swanson 
    7 months ago
    $35
  2. Margaret Backer 
    1 year ago
    $1,000
  3. Jen & Martin Schreier 
    2 years ago
    $500
  4. MilliporeSigma  
    2 years ago
    $1,000
  5. Anne & Michael Zdrodowski 
    2 years ago
    $150
  6. Nancy & Brian Askari 
    2 years ago
    $50
  7. Karin Barrows 
    2 years ago
    $50
  8. Verizon Founation  
    2 years ago
    $100
  9. Carrie & Avram Rossenfeld 
    2 years ago
    $25
  10. Lorraine Serr-Urquhart 
    2 years ago
    $100
  11. Jennifer Glaser 
    2 years ago
    $25
  12. Nicole Ahrens  
    2 years ago
    $50
  13. Anonymous 
    2 years ago
    $50
  14. Dan Hoskins 
    2 years ago
    $500
  15. Margaret Velardo  
    2 years ago
    $100
  16. Ed and Gabrielle Nield 
    2 years ago
    $100
  17. Debbie and Terry Loughran 
    2 years ago
    $100
  18. Meryl King 
    2 years ago
    $50
  19. Libby Smith 
    2 years ago
    $100
  20. Joanne and Michael Rykowski  
    2 years ago
    $100
  21. Joanne Stress  
    2 years ago
    $20
  22. Melanie Landers 
    2 years ago
    $50
  23. Zachary Lefevre 
    2 years ago
    $10
  24. Emily Backer 
    2 years ago
    $50
  25. Christy Sauer 
    2 years ago
    $100
  26. Lynn Schenk 
    2 years ago
    $100
  27. Abisola Oseni 
    2 years ago
    $100
  28. Mary Jo Uebelhor 
    2 years ago
    $500
  29. Joseph Backer 
    2 years ago
    $500
  30. Jennifer Lucas 
    2 years ago
    $100
  31. Laura and Kelly Roggenkamp 
    2 years ago
    $100
  32. Catherine and Joe Hall 
    2 years ago
    $100
  33. Linda Sirugo 
    2 years ago
    $1,000
  34. Laura Backer 
    2 years ago
    $2,000
  35. Monique Beal 
    2 years ago
    $50
  36. Marjorie Flores 
    2 years ago
    $100
  37. Julie Putnam 
    2 years ago
    $20
  38. Anonymous 
    2 years ago
    $50
  39. John Greener 
    2 years ago
    $250
  40. John Ferry 
    2 years ago
    $100
  41. Anonymous 
    2 years ago
    $500
  42. Tracy Kaslander Wohning 
    2 years ago
    $100
  43. Eileen Anderson  
    2 years ago
    $100
  44. Anonymous 
    2 years ago
    $30
  45. Allie and Randi Brissman 
    2 years ago
    $100
  46. Robert & Patricia Swanson 
    2 years ago
    $100
  47. Lisa Gil 
    2 years ago
    $50
  48. Megan Schaeffer 
    2 years ago
    $100
  49. Katherine Mruczkowski 
    2 years ago
    $50
  50. Karri Smith 
    2 years ago
    $25
  51. Elyse Hovanesian 
    2 years ago
    $50
  52. Evelyn Demirjian 
    2 years ago
    $30
  53. Anthony Uebelhor 
    2 years ago
    $250
  54. Jolie Raymond 
    2 years ago
    $25
  55. Grant Gilmore 
    2 years ago
    $100
  56. Steve Bordley 
    2 years ago
    $100
  57. Julia Sofo 
    2 years ago
    $20
  58. Mary Beyer 
    2 years ago
    $100
  59. Anonymous 
    2 years ago
    $10
  60. Patrick Dodd 
    2 years ago
    $250
  61. Joan Tennison 
    2 years ago
    $100
  62. Adam Levitsky 
    2 years ago
    $100
  63. Grace Backer 
    2 years ago
    $30
  64. Buck and Deborah Burt 
    2 years ago
    $50
  65. Marlene Hein 
    2 years ago
    $50
  66. Tina Van Hamersveld 
    2 years ago
    $20
  67. U Kelley Berman 
    2 years ago
    $50
  68. Barbars Donadio 
    2 years ago
    $50
  69. Christine Vanderpool 
    2 years ago
    $50
  70. Thomas Buechler 
    2 years ago
    $100
  71. Daniel Backer 
    2 years ago
    $100
  72. Annette Crawford 
    2 years ago
    $500
  73. Kim Hedges 
    2 years ago
    $100
  74. Karen & Jeff Parkhill 
    2 years ago
    $250
  75. Andrew Dodd 
    2 years ago
    $250
  76. Alexandra Rykowski 
    2 years ago
    $50
  77. bruce boeger 
    2 years ago
    $200
  78. Janet Schenker 
    2 years ago
    $200
  79. Katarina Uebelhor 
    2 years ago
    $50
  80. Michael Zdrodowski 
    2 years ago
    $100
  81. Matthew Holden 
    2 years ago
    $500
  82. Dan Bolstrum 
    2 years ago
    $100
  83. Sandra McGill 
    2 years ago
    $300
  84. Barry Grigsby 
    2 years ago
    $100
  85. Gregory Williams 
    2 years ago
    $200
  86. Angela Bates 
    2 years ago
    $100
  87. Sarah & Lindsay Knapp 
    2 years ago
    $1,000
  88. Karen & Jeff Parkhill 
    2 years ago
    $250
  89. Sue and Rich Mirman 
    2 years ago
    $1,000
  90. Lucile Lynch 
    2 years ago
    $250
  91. Diana Miller 
    2 years ago
    $100
  92. Uncle Glen Schreitmueller 
    2 years ago
    $1,000
  93. Brian Gonzalez 
    2 years ago
    $500
  94. Jane Woltman 
    2 years ago
    $100
  95. George Backer 
    2 years ago
    $1,000
  96. David Backer 
    2 years ago
    $10,000
  97. Hollianne & Shaun Uebelhor 
    2 years ago
    $750

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