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 (93)

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

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