94% dos pacientes com terapia genética com adrenoleucodistrofia cerebral mantiveram a função neurológica seis anos após o tratamento. 94% of gene therapy patients with cerebral adrenoleukodystrophy maintained neurological function six years post-treatment.

A terapia genética com elivaldogene autotemcel (eli-cel) mostra resultados promissores a longo prazo para a adrenoleucodistrofia cerebral (CALD), uma doença genética grave que afeta meninos jovens. Gene therapy with elivaldogene autotemcel (eli-cel) shows promising long-term results for cerebral adrenoleukodystrophy (CALD), a severe genetic disorder affecting young boys. Seis anos após o tratamento, 94% dos pacientes mantiveram a função neurológica e mais de 80% evitaram a incapacidade maior. Six years post-treatment, 94% of patients maintained neurological function, and over 80% avoided major disability. No entanto, os riscos de cânceres hematológicos, incluindo síndrome mielodisplásica e leucemia mielóide aguda, foram observados. However, risks of hematologic cancers, including myelodysplastic syndrome and acute myeloid leukemia, were noted. Os pesquisadores continuarão a refinar os protocolos de tratamento e investigar esses riscos. Researchers will continue to refine treatment protocols and investigate these risks.