The FDA Approves Rocket Pharmaceuticals' Kresladi For Pediatric Patients With Severe Leukocyte Adhesion Deficiency-I

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First FDA-approved gene therapy for children with severe LAD-I due to biallelic variants in ITGB2
Severe LAD-I is an ultra-rare, life-threatening pediatric genetic immunodeficiency characterized by recurrent infections and high early-childhood mortality without treatment
FDA grants Rare Pediatric Disease Priority Review Voucher

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The FDA Approves Rocket Pharmaceuticals&#39; Kresladi For Pediatric Patients With Severe Leukocyte Adhesion Deficiency-I