Gene therapy shows long-term benefit for patients with Cerebral adrenoleukodystrophy (CALD)
Overview:
In a study published in The New England Journal of Medicine, lentiviral gene therapy using elivaldogene autotemcel (eli-cel) showed promising results in treating cerebral adrenoleukodystrophy (CALD) in boys. Over a 24-month period, 91% of participants survived without major functional disabilities, and 94% showed stable neurologic function over a median of 6 years. While the therapy was largely effective, one patient developed myelodysplastic syndrome, highlighting an ongoing risk associated with viral vector therapies. For more details, you can access the full study here.
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