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Ultragenyx Pharmaceutical Inc.
5 posts
Ultragenyx adds sixth FDA-approved product as FAYUVI targets Sanfilippo syndrome
Ultragenyx Pharmaceutical Inc. has secured full FDA approval for FAYUVI, a $3.95 million one-time gene therapy for Sanfilippo syndrome type A, giving the rare-disease company a major commercial catalyst weeks after another program wiped 44% from its share price.
Ultragenyx (Nasdaq: RARE) wins FDA approval for $2.7m Genglycos gene therapy
Genglycos becomes the first approved treatment for glycogen storage disease type Ia, but accelerated approval rests on reduced cornstarch use as a surrogate endpoint and requires confirmatory evidence.
Is Mereo BioPharma now a hidden EU rare disease play with Ultragenyx exposure?
Mereo BioPharma holds EU and UK rights to setrusumab. Can upcoming Phase 3 data make it a hidden rare disease winner in 2025?
Can final-year OI data rescue Ultragenyx’s $245M pipeline gamble on setrusumab?
Ultragenyx’s setrusumab trial didn’t stop early—but can final data still unlock $245M in milestones and rescue biotech investor confidence?
Ultragenyx stock plummets 25% after Phase 3 OI trial continues without early stop
Ultragenyx stock plunged 25% as the UX143 Phase 3 trial for osteogenesis imperfecta proceeds without early stop. Find out what this means for investors.