94% of gene therapy patients with cerebral adrenoleukodystrophy maintained neurological function six years post-treatment.

Gene therapy with elivaldogene autotemcel (eli-cel) shows promising long-term results for cerebral adrenoleukodystrophy (CALD), a severe genetic disorder affecting young boys. Six years post-treatment, 94% of patients maintained neurological function, and over 80% avoided major disability. However, risks of hematologic cancers, including myelodysplastic syndrome and acute myeloid leukemia, were noted. Researchers will continue to refine treatment protocols and investigate these risks.

October 09, 2024
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