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- Beacon Therapeutics reports positive pivotal trial results for X-linked retinitis pigmentosa gene therapyBeacon Therapeutics said its gene therapy for X-linked retinitis pigmentosa (XLRP) met the primary endpoint in a phase 2/3 Vista study. The trial enrolled 85 male patients and compared two dose lev…
- Ultragenyx receives FDA approval for Fayuvi, first therapy for Sanfilippo syndrome type A, priced at $3.95 MThe FDA has approved Ultragenyx's gene therapy rebisufligene etisparvovec, to be sold under the brand name Fayuvi, for the treatment of Sanfilippo syndrome type A, an ultra-rare progressive neurolo…BioSpace · 1mo agoApprovalPositive