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This chapter discusses trials in which gene therapy is used to treat Pompe disease. The trials cover gene therapy use in both patients with late onset and infantile onset Pompe disease. The trials also cover the efficacy and safety of gene therapy use in both late onset and infantile onset Pompe disease.

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Pompe Disease

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  • 发表日期：

    2025年

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  • Jeffrey N. Weiss

摘要

This chapter discusses trials in which gene therapy is used to treat Pompe disease. The trials cover gene therapy use in both patients with late onset and infantile onset Pompe disease. The trials also cover the efficacy and safety of gene therapy use in both late onset and infantile onset Pompe disease.