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Primary Immunodeficiencies

Activated Phosphoinositide 3-Kinase Delta Syndrome Leukocyte Adhesion Deficiency WHIM Syndrome

FDA Grants Accelerated Approval to Kresladi, First Gene Therapy for Severe Pediatric LAD-1

The one-time autologous treatment won conditional clearance on biomarker gains, requiring long‑term confirmation of benefit.

CHMP Backs Xolremdi for WHIM Syndrome Under Exceptional Circumstances