Gene Therapy for Infantile-Onset Pompe's Disease

  • Parenti, Giancarlo M.D.
  • Brunetti-Pierri, Nicola M.D.

  • 1Department of Translational Medicine, University of Naples Federico II, Naples, Italy

    2Telethon Institute of Genetics and Medicine, Pozzuoli, Italy

    3Scuola Superiore Meridionale, Genomics and Experimental Medicine Program, University of Naples Federico II, Naples, Italy

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New England Journal of Medicine 392(24):p 2477-2478, June 26, 2025. | DOI: 10.1056/NEJMe2506264
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