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    • #15937

      Gene Therapy for Muscular Dystrophy

      “Conner Curran was 4 years old when he was diagnosed with Duchenne muscular dystrophy, a genetic disease that causes muscles to waste away. Five years later, Conner is not just walking, but running faster than ever, thanks to an experimental gene therapy that took more than 30 years to develop. Conner was the first child to receive the treatment — a single infusion designed to fix the genetic mutation that was gradually causing his muscles cells to die. The treatment can’t bring back the cells he’s lost (he remains smaller and weaker than his twin brother, Kyle), but it has allowed the muscle cells he still has to function better.”

      What do you think about gene therapy? Does this article give you hope for other forms of MD?

    • #17205
      Barbara la saracina
      Participant

      Are they doing clinical traiks with this gene therapy on young dechenne patients

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