FDA approves gene therapy for MPS IIIA - Poll - MDSpire
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FDA approves gene therapy for MPS IIIA

The one-time intravenous treatment uses adeno-associated virus serotype 9 to deliver a functional copy of the SGSH gene.

  • By

  • Kathryn Wighton

  • September 18, 2026

  • 2 min

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FDA approves gene therapy for MPS IIIA

  • by Kathryn Wighton

  • September 18, 2026

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