An experimental gene therapy has successfully treated more than 60 children born with ADA-SCID, reversing their immune failure in one once-only treatment. The breakthrough, with up to 12 years of stable follow-up, charts a new direction in rare-disease medicine.
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Ground-breaking therapy saves over 60 children from deadly condition

EL PAIS

An experimental gene therapy has successfully treated more than 60 children born with ADA-SCID, reversing their immune failure in one once-only treatment. The breakthrough, with up to 12 years of stable follow-up, charts a new direction in rare-disease medicine.

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