La FDA ha aprobado Fayuvi (rebisufligene etisparvovec-hopf), primera terapia génica aprobada en la historia para el Síndrome de Sanfilippo Tipo A (MPS IIIA), una enfermedad lisosomal neurodegenerativa ultra rara que causa un deterioro neurológico infantil progresivo.

FDA Approves First Gene Therapy for Pediatric Patients with Sanfilippo Syndrome Type A Innovative gene therapy offers a new option for children with a rare, progressive neurodevelopmental disease https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-pediatric-patients-sanfilippo-syndrome-type?utm_medium=email&_hsenc=p2ANqtz-_lY-dmunKpHnGU2uHMQXJKQY8mhXLkovpXf6wYj2-MzRFo9WC-vg8j6Tp6ZXk_hCtKC-0dHInsLEnGVN9XcCYyTn4QuA&_hsmi=146512528&utm_content=146523058&utm_source=hs_email