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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Una terapia pionera salva a más de 60 niños con enfermedad letal

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Una terapia génica experimental trató con éxito a más de 60 niños con ADA-SCID, invirtiendo su fallo inmunitario con una única intervención. Los resultados, con hasta 12 años de seguimiento estable, abren una nueva vía en el tratamiento de enfermedades raras.

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