FDA OKs Therapy for Deadly Pediatric Immune Disorder

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Source: MedPage Today

Original: https://www.medpagetoday.com/genetics/generalgenetics/120525...

Published: Fri, 27 Mar 2026 12:37:43 -0400

The FDA has approved marnetegragene autotemcel (Kresladi) as the first gene therapy to treat children with severe leukocyte adhesion deficiency type I (LAD-I). LAD-I is a rare inherited immunodeficiency. The approval was granted in an accelerated procedure. Kresladi is intended for patients aged one month and older. This is the first gene therapy approved for this particular disorder.