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First Spanish Child Cured of Wiskott-Aldrich Syndrome via Gene Therapy
Five-year-old Javier, diagnosed with Wiskott-Aldrich syndrome at six months old, is the first child in Spain cured using gene therapy; this case highlights the potential of gene therapy and challenges of accessing innovative treatments for rare diseases affecting less than 1 in 250,000 male births.
First Spanish Child Cured of Wiskott-Aldrich Syndrome via Gene Therapy
Five-year-old Javier, diagnosed with Wiskott-Aldrich syndrome at six months old, is the first child in Spain cured using gene therapy; this case highlights the potential of gene therapy and challenges of accessing innovative treatments for rare diseases affecting less than 1 in 250,000 male births.
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