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Rocky Ford Family Raising Money for Possible Cure to Son’s Rare, Terminal Disease

  • Experimental gene therapy for Fabry disease is saving treatment costs for patients, as reported by West.
  • Three men treated for Fabry disease discontinued enzyme-replacement therapy, which costs about $300,000 annually, after starting gene therapy.
  • Larger studies are needed before gene therapy can become a conventional treatment due to existing therapy's high costs and patient burden.
  • The goal is to expand the study to include 25 to 30 patients over a two- to three-year period, according to West.
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Ariel was born with a serious genetic disease, her father Dr. Shternin decided not to give up and find a cure • At the end of the journey, he managed to reach a breakthrough - "It sounds a bit like science fiction - the drug simply cuts the damaged DNA" • Her father told about the moment he discovered the disease: "As a doctor, I got to tell the news about children with diseases, but as a father - the ground falls away from under your feet" • To…

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KRDO broke the news in Colorado Springs, United States on Friday, July 4, 2025.
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