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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…
- Clinical trial enrollment crisis threatens billions in revenue and patient accessClinical trials are struggling with enrollment, with about 80% missing timelines and less than 5% of eligible adult cancer patients participating. Delays have huge financial impact; each day a pha…