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Ultragenyx Wins Sanfilippo Type A Approval & Xenon Pauses New Psychiatry Enrollment
Description
Ultragenyx's Fayuvi approval opens a first treatment path for eligible children with Sanfilippo syndrome type A, with preserved function and center access defining the boundary. Alex and Maya examine Xenon's azetukalner epilepsy filing and separate pause in new psychiatry enrollment, plus Roche's Phase 3 CELESTIMO endpoint result for Lunsumio in follicular lymphoma. They also cover Lisata's Marea acquisition and financing; Roche's Dualitas deal; Longeveron's laromestrocel miss in hypoplastic left heart syndrome; Bayer's Kerendia expansion in type 1 diabetes kidney disease; NICE's Enhertu access recommendation in England; and Aquestive's Anaphylm resubmission.
Alex Mercer and Maya Patel host. Get the daily rundown in your inbox — subscribe at https://thepharmacloseout.com.