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Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy

9 Oct 2024

Abstract excerpt

BACKGROUND: complementary DNA, is being tested in persons with cerebral adrenoleukodystrophy. METHODS: In a phase 2-3 study, we evaluated the efficacy and safety of eli-cel therapy in boys with early-stage cerebral adrenoleukodystrophy and evidence of active inflammation on magnetic resonance imaging (MRI). The primary efficacy end point was survival without any of six major functional disabilities at month 24....

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PMID: 39383459
DOI: 10.1056/nejmoa2400442

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