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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Bahnbrechende Therapie rettet über 60 Kinder vor tödlicher Erkrankung

EL PAIS

Eine experimentelle Gentherapie behandelte erfolgreich mehr als 60 Kinder mit ADA-SCID und kehrte ihren Immunversagen mit nur einer einzigen Anwendung um. Mit bis zu zwölf Jahren stabiler Nachbeobachtung markiert dieser Fortschritt eine neue Ära in der Therapie seltener Erkrankungen.

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