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Story summary
- An investigational gene therapy for adenosine deaminase severe combined immunodeficiency (ADA-SCID) showed significant clinical efficacy and safety.
- The study of 62 children reported 100% overall survival and 95% event-free survival after a median follow-up of 7.5 years, in the New England Journal of Medicine.
- Most participants had previously received standard treatments.
- High manufacturing costs limit commercial viability, despite potential long-term savings from reduced hospital stays and complications.
