在基因治疗中,有94%的脑上腺核缩症患者在治疗后6年内保持神经功能. 94% of gene therapy patients with cerebral adrenoleukodystrophy maintained neurological function six years post-treatment.

elivaldogene autotemcel （eli-cel） 的基因疗法对脑肾上腺脑白质营养不良 （CALD） 显示出有希望的长期结果，这是一种影响年轻男孩的严重遗传疾病。 Gene therapy with elivaldogene autotemcel (eli-cel) shows promising long-term results for cerebral adrenoleukodystrophy (CALD), a severe genetic disorder affecting young boys. 6年后治疗,94%的病人保持神经功能,80%以上的病人避免了重大残疾。 Six years post-treatment, 94% of patients maintained neurological function, and over 80% avoided major disability. 然而,还发现了血液癌症的风险,包括骨髓位综合征和急性骨髓性白血病. However, risks of hematologic cancers, including myelodysplastic syndrome and acute myeloid leukemia, were noted. 研究人员将继续完善治疗规程并调查这些风险。 Researchers will continue to refine treatment protocols and investigate these risks.