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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…
- England's NHS to fund AstraZeneca's Enhertu for metastatic breast cancer after U.K.-U.S. pricing pactEnglish health authorities have agreed to provide AstraZeneca's breast cancer drug Enhertu through the NHS, ending a multi-year dispute over its availability. The decision will make the drug acces…