Health
FDA Approves New Gene Therapy for Rare Disease
The FDA has approved a new gene therapy for Sanfilippo syndrome type A, an ultra-rare disease. The treatment is seen as a significant breakthrough in managing this condition.
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STAT News
The FDA approved a new gene therapy for Sanfilippo syndrome type A, an ultra-rare disease sometimes known as childhood Alzheimer’s.
2026-09-17 19:55 UTC