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FDA批准Rocket Pharmaceuticals的Kresladi用于患有严重白细胞粘附缺陷的儿科患者-I

2026-03-27 19:06

Rocket制药(RCKT) 0

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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