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FDA Approves Gene Therapy for Severe Pediatric LAD-I

Approval expands treatment option for a rare pediatric immunodeficiency

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  • 1

    KRESLADI approved for severe leukocyte adhesion deficiency-I.

  • 2

    ITGB2 gene variants lead to high mortality in infants.

  • 3

    Incidence in the US: 1 in 100,000 to 1 in 200,

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    KRESLADI is an autologous gene therapy.

  • 5

    Risks include serious infections and potential oncogenesis.

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    Approval contingent on long-term benefit verification.

  • 7

    A Rare Pediatric Disease Priority Review Voucher was awarded.

Original Source(s)

FDA Approves Gene Therapy for Severe Pediatric LAD-I

  • by Kathryn Wighton

  • March 27, 2026

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