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 (95)

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

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