Seeking Alpha

Ultragenyx Announces Plans to File for Accelerated Approval of UX111 for the Treatment of Sanfilippo Syndrome Type A (MPS IIIA)

NOVATO, Calif., June 12, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (RARE) today announced that the company held a successful meeting with the U.S. Food and Drug Administration (FDA or ...
Seeking Alpha

Ultragenyx Submits Biologics License Application to the U.S. FDA for UX111 AAV Gene Therapy for the Treatment of Sanfilippo Syndrome Type A (MPS IIIA)

NOVATO, Calif., Dec. 19, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (RARE) today announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration ...
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Ultragenyx’s gene therapy shows sustained improvements in MPS IIIA

Ultragenyx Pharmaceutical’s gene therapy has shown durable improvements in patients with Sanfilippo syndrome Type A (MPS IIIA), a rare neurodegenerative disorder. A Phase I/II/III trial (NCT02716246) ...
WNCT

Ultragenyx Announces FDA Acceptance and Priority Review of the Biologics License Application (BLA) for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)

NOVATO, Calif., Feb. 18, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced the U.S. Food and Drug Administration (FDA or the Agency) has accepted for review the ...
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Ultragenyx’s MPS VII Study: A Closer Look at Market Implications

Ultragenyx Pharmaceutical, Inc. ((RARE)) announced an update on their ongoing clinical study. Take advantage of TipRanks Premium at 50% off! Unlock powerful investing tools, advanced data, and expert ...
Nasdaq

Andelyn Biosciences Selected by Ultragenyx for Late-Stage Process Performance Qualification (PPQ) Manufacturing of Novel Gene Therapy for Sanfilippo Syndrome

COLUMBUS, Ohio, Dec. 18, 2023 /PRNewswire/ -- Andelyn Biosciences, Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), announced ...