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 (96)

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

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