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- FDA Approves First Gene Therapy for Pediatric Patients with Sanfilippo Syndrome Type AThe U.S. Food and Drug Administration today approved Fayuvi (rebisufligene etisparvovec-hopf), the first treatment for pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA), also known…
- Roche's Lunsumio clears Phase 3 hurdle, paving way for broader label in indolent lymphomaRoche announced that its T cell engager Lunsumio successfully met the primary endpoints in a Phase 3 study of a slow growing blood cancer. The trial, conducted in patients with indolent lymphoma,…