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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.

EU Seals Provisional Overhaul of Drug Rules to Speed Access and Spur Innovation

Sarepta Shares Plunge After ESSENCE Phase 3 Miss in Duchenne Trial

Pfizer–Trump Drug-Pricing Deal and ‘TrumpRx’ Plan Jolt Health-Care Stocks Higher

Rapport Seizure Drug Posts Strong Phase 2 Signal as Company Plans Phase 3

Vertex Halts VX-993 Acute Pain Program After Phase 2 Trial Failure