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.

October 09, 2024

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