This child was treated for a rare genetic disease while still in the womb

A toddler girl is flourishing after receiving treatment for a rare genetic disease. In a first for this disease, she received that treatment before she was even born.

Sixteen-month-old Ayla has infantile-onset Pompe disease — a genetic disorder that can cause organ damage that begins before birth. Babies born with Pompe have enlarged hearts and weak muscles. If left untreated, most infants die before they turn 2. Treatment typically begins after birth, but that tactic doesn’t prevent the irreversible, and potentially deadly, organ damage that happens in utero.

Ayla received treatment while still in the womb as part of an early-stage clinical trial. Today, the toddler has a normal

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