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Mom Told Daughter Would Be ‘Locked in Her Body’—She Refused To Give Up

Mom Told Daughter Would Be ‘Locked in Her Body’—She Refused To Give Up

Summary

A four-year-old girl named Harlow Prado, who has a rare brain disorder, received a groundbreaking personalized treatment that could help her walk. Her condition, TUBB4A-related leukodystrophy, affects her ability to move and requires regular therapy. On October 15, she became the first girl in the world to receive this type of gene therapy.

Key Facts

  • Harlow Prado has a rare brain disorder called TUBB4A-related leukodystrophy.
  • This disorder affects the myelin, which is the protective covering of nerve cells.
  • Harlow has needed a walker and daily therapy sessions since birth.
  • TUBB4A leukodystrophy is often misdiagnosed and needs genetic testing for proper identification.
  • There is no known cure for the disorder, and its symptoms can lead to muscle issues and paralysis.
  • A nonprofit organization called n-Lorem developed a treatment specifically for Harlow.
  • Harlow's family raised $100,000 through crowdfunding to cover treatment costs.
  • Harlow received her first dose of gene therapy on October 15, making her the first girl to get this treatment.
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