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 (67)

  1. Laura and Kelly Roggenkamp 
    8 hours ago
    $100
  2. Catherine and Joe Hall 
    1 day ago
    $100
  3. Linda Sirugo 
    1 day ago
    $1,000
  4. Laura Backer 
    2 days ago
    $2,000
  5. Monique Beal 
    2 days ago
    $50
  6. Marjorie Flores 
    3 days ago
    $100
  7. Julie Putnam 
    3 days ago
    $20
  8. Anonymous 
    3 days ago
    $50
  9. John Greener 
    3 days ago
    $250
  10. John Ferry 
    4 days ago
    $100
  11. Anonymous 
    4 days ago
    $500
  12. Tracy Kaslander Wohning 
    4 days ago
    $100
  13. Eileen Anderson  
    4 days ago
    $100
  14. Anonymous 
    4 days ago
    $30
  15. Allie and Randi Brissman 
    4 days ago
    $100
  16. Robert & Patricia Swanson 
    4 days ago
    $100
  17. Lisa Gil 
    4 days ago
    $50
  18. Megan Schaeffer 
    4 days ago
    $100
  19. Katherine Mruczkowski 
    5 days ago
    $50
  20. Karri Smith 
    5 days ago
    $25
  21. Elyse Hovanesian 
    5 days ago
    $50
  22. Evelyn Demirjian 
    5 days ago
    $30
  23. Anthony Uebelhor 
    5 days ago
    $250
  24. Jolie Raymond 
    5 days ago
    $25
  25. Grant Gilmore 
    5 days ago
    $100
  26. Steve Bordley 
    5 days ago
    $100
  27. Julia Sofo 
    5 days ago
    $20
  28. Mary Beyer 
    5 days ago
    $100
  29. Anonymous 
    5 days ago
    $10
  30. Patrick Dodd 
    5 days ago
    $250
  31. Joan Tennison 
    5 days ago
    $100
  32. Adam Levitsky 
    5 days ago
    $100
  33. Grace Backer 
    5 days ago
    $30
  34. Buck and Deborah Burt 
    5 days ago
    $50
  35. Marlene Hein 
    5 days ago
    $50
  36. Tina Van Hamersveld 
    5 days ago
    $20
  37. U Kelley Berman 
    5 days ago
    $50
  38. Barbars Donadio 
    5 days ago
    $50
  39. Christine Vanderpool 
    5 days ago
    $50
  40. Thomas Buechler 
    5 days ago
    $100
  41. Daniel Backer 
    1 week ago
    $100
  42. Annette Crawford 
    1 week ago
    $500
  43. Kim Hedges 
    1 week ago
    $100
  44. Karen & Jeff Parkhill 
    2 weeks ago
    $250
  45. Andrew Dodd 
    2 weeks ago
    $250
  46. Alexandra Rykowski 
    2 weeks ago
    $50
  47. bruce boeger 
    3 weeks ago
    $200
  48. Janet Schenker 
    3 weeks ago
    $200
  49. Katarina Uebelhor 
    4 weeks ago
    $50
  50. Michael Zdrodowski 
    4 weeks ago
    $100
  51. Matthew Holden 
    1 month ago
    $500
  52. Dan Bolstrum 
    1 month ago
    $100
  53. Sandra McGill 
    1 month ago
    $300
  54. Barry Grigsby 
    2 months ago
    $100
  55. Gregory Williams 
    2 months ago
    $200
  56. Angela Bates 
    2 months ago
    $100
  57. Sarah & Lindsay Knapp 
    2 months ago
    $1,000
  58. Karen & Jeff Parkhill 
    2 months ago
    $250
  59. Sue and Rich Mirman 
    2 months ago
    $1,000
  60. Lucile Lynch 
    2 months ago
    $250
  61. Diana Miller 
    2 months ago
    $100
  62. Uncle Glen Schreitmueller 
    2 months ago
    $1,000
  63. Brian Gonzalez 
    2 months ago
    $500
  64. Jane Woltman 
    2 months ago
    $100
  65. George Backer 
    2 months ago
    $1,000
  66. David Backer 
    2 months ago
    $10,000
  67. Hollianne & Shaun Uebelhor 
    4 weeks ago
    $750

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