Without treatment, those with the more severe form of illness may die before their second birthday
  • Hotznplotzn@lemmy.sdf.org
    5·
    1 day ago

    I read of a similar treatment for another disease two years ago and found the report. That’s absolutely amazing.

    In a first, doctors treat fatal genetic disease before birth (November 2022)

    A toddler is thriving after doctors in the U.S. and Canada used a novel technique to treat her before she was born for a rare genetic disease that caused the deaths of two of her sisters.

    Ayla Bashir, a 16-month-old from Ottawa, Ontario, is the first child treated as fetus for Pompe disease, an inherited and often fatal disorder in which the body fails to make some or all of a crucial protein.