In a first, a fatal enzyme deficiency is treated in the womb

To protect this child from the same genetic disease that killed two older siblings, treating her as soon as she was born might only work so well, the doctors knew. So they dialed back the therapeutic clock, delivering the medication to her as a fetus.

Now 16 months old, Ayla appears totally healthy. She still requires weekly doses of the medication, an enzyme that she can’t produce on her own, but she has no symptoms of the condition she inherited, the most serious form of the rare Pompe disease. Her heart is strong, and she started walking at a typical 11 months old.

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