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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Une thérapie novatrice sauve plus de 60 enfants d’une maladie mortelle

EL PAIS

Une thérapie génique expérimentale a permis de traiter avec succès plus de 60 enfants atteints d’ADA-SCID, rétablissant leur système immunitaire après une seule administration. Suivis jusqu’à 12 ans, ces résultats ouvrent une nouvelle ère dans le traitement des maladies rares.

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