Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy
The study publicshed in the New England Journal of Medicine titled “Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy” investigates the long-term safety of the gene therapy elivaldogene autotemcel (eli-cel) in patients with cerebral adrenoleukodystrophy (CALD). The therapy modifies patients’ own blood-forming stem cells to include a functional gene to manage CALD. However, 7 of …
Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy Read More »