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new data highlights cancer risks in gene therapy for childhood brain disorder

New data reveal a cancer risk associated with Skysona, a gene therapy for cerebral adrenoleukodystrophy (CALD), with seven cases of blood malignancies reported among 67 treated boys. While the therapy shows promise, concerns about its safety persist, prompting careful consideration by physicians and families. Bluebird bio, the therapy's developer, emphasizes patient well-being and informed decision-making amid ongoing scrutiny.
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