The FDA Approves Rocket Pharmaceuticals' Kresladi For Pediatric Patients With Severe Leukocyte Adhesion Deficiency-I
Benzinga Newsdesk 7 months ago
BiotechNewsHealth CareFDAGeneral
First FDA-approved gene therapy for children with severe LAD-I due to biallelic variants in ITGB2Severe LAD-I is an ultra-rare, life-threatening pediatric genetic immunodeficiency characterized by recurrent infections
- 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