FDA Approves Gene Therapy for Severe Pediatric LAD-I - Top-Institutions - MDSpire

FDA Approves Gene Therapy for Severe Pediatric LAD-I

Approval expands treatment option for a rare pediatric immunodeficiency

  • By

  • Kathryn Wighton

  • March 27, 2026

  • 2 min

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Top Institutions in Pediatrics

Brief introduction explaining scope and methodology.

  • #1

    University of California, Los Angeles (UCLA)
    University of California, Los Angeles (UCLA)

    Los Angeles, CA

    Key Differentiators

    • Pediatric Immunology
    • Gene Therapy
    • Hematopoietic Stem Cell Transplantation

  • #2

    National Institutes of Health (NIH) - National Institute of Allergy and Infectious Diseases (NIAID)
    National Institutes of Health (NIH) - National Institute of Allergy and Infectious Diseases (NIAID)

    Bethesda, MD

    Key Differentiators

    • Immunology
    • Gene Therapy
    • Rare Diseases

  • #3

    St. Jude Children's Research Hospital
    St. Jude Children's Research Hospital

    Memphis, TN

    Key Differentiators

    • Pediatric Hematology/Oncology
    • Gene Therapy
    • Immunodeficiency

  • #4

    Boston Children's Hospital
    Boston Children's Hospital

    Boston, MA

    Key Differentiators

    • Pediatric Immunology
    • Gene Therapy
    • Stem Cell Transplantation

Original Source(s)

FDA Approves Gene Therapy for Severe Pediatric LAD-I

  • by Kathryn Wighton

  • March 27, 2026

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