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  1. Jun 12, 2024 · Ultragenyx, Mereo drug reduces fractures in bone disorder study. New data show the experimental treatment’s effect on fractures and bone density held up over time, boosting the drug’s prospects ahead of a Phase 3 readout.

  2. Jun 11, 2024 · The partnership with Ultragenyx includes potential additional milestone payments of up to $245 million and royalties to Mereo on commercial sales in Ultragenyx territories.

  3. Jun 13, 2024 · With the FDA agreeing a biomarker is a reasonable surrogate endpoint, Ultragenyx is preparing to file for approval of its Sanfilippo gene therapy.

  4. Jun 12, 2024 · Ultragenyx and Mereo BioPharma have broken off another piece of brittle bone data, linking their drug candidate to sustained reductions in fracture rates in a midphase clinical trial.

  5. Jun 12, 2024 · Agreement reached with FDA that cerebral spinal fluid (CSF) heparan sulfate (HS) can be used as a reasonable surrogate endpoint for accelerated approval. Data from existing clinical studies appear sufficient for a biologics license application (BLA) submission.

  6. Jun 12, 2024 · About Ultragenyx. Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultrarare genetic diseases.

  7. Jun 13, 2024 · Ultragenyx Pharmaceutical Inc. RARE announced that it is planning to seek accelerated approval for its AAV gene therapy candidate UX111, which is being developed for the treatment of Sanfilippo...