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Gene Mutations

BRCA1 and BRCA2 NPM1 and FLT3-ITD KRAS and EGFR Mutations Mitochondrial Diseases PTEN Mutation ITGB2 Gene EGFR Mutations

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.

Proteomic Test Cuts Childhood Rare Disease Diagnosis to Under Three Days