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Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) news

Ultragenyx Pharmaceutical Inc. news releases on 6ix. Latest, Oct 2, 2026: Ultragenyx Announces Marketing Authorisation Application (MAA) Submission…

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) news

Press releases & updates

Ultragenyx Announces Marketing Authorisation Application (MAA) Submission to the European Medicines Agency (EMA) for the First Investigational Gene Therapy for MPS IIIA (Sanfilippo Syndrome Type A)

Ultragenyx Announces Marketing Authorisation Application (MAA) Submission to the European Medicines Agency (EMA) for the First Investigational Gene Therapy for MPS IIIA (Sanfilippo Syndrome Type A)

Ultragenyx Announces Approval of FAYUVI Gene Therapy, the First-Ever FDA-Approved Treatment for Sanfilippo Syndrome Type A

Ultragenyx Announces Approval of FAYUVI™ Gene Therapy, the First-Ever FDA-Approved Treatment for Sanfilippo Syndrome Type A (MPS IIIA)

Ultragenyx Announces Phase 3 Aspire results in Angelman Syndrome

Ultragenyx Announces the Publication of a Successful 96-Week Randomized, Placebo-Controlled Trial with Crossover Treatment of GENGLYCOS™ (also known as DTX401) AAV Gene Therapy in GSDIa in The Journal of Inherited Metabolic Disease

Ultragenyx Announces U.S. FDA Approval of GENGLYCOS Gene Therapy, the First-Ever FDA-Approved Treatment Designed to Treat the Underlying Cause of Glycogen Storage Disease Type Ia (GSDIa)

Ultragenyx Announces U.S. FDA Approval of GENGLYCOS™ Gene Therapy, the First-Ever FDA-Approved Treatment Designed to Treat the Underlying Cause of Glycogen Storage Disease Type Ia (GSDIa)

Ultragenyx Reports Second Quarter 2026 Financial Results and Corporate Update

Ultragenyx to Host Conference Call for Second Quarter 2026 Financial Results and Corporate Update

Ultragenyx Releases 2025 Impact Report Emphasizing Commitment to Rare Disease Patients, Innovation, and Global Impact

Ultragenyx to Participate at Goldman Sachs 47th Annual Global Healthcare Conference

Ultragenyx to Participate at Bank of America’s 2026 Healthcare Conference

Ultragenyx Reports First Quarter 2026 Financial Results and Corporate Update

Ultragenyx to Host Conference Call for First Quarter 2026 Financial Results and Corporate Update

Ultragenyx Announces U.S. FDA Acceptance of BLA Resubmission for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)

Ultragenyx Announces FDA Clearance of Investigational New Drug Application for UX016, a Sialic Acid Prodrug for the Treatment of GNE Myopathy

Ultragenyx Announces FDA Clearance of Investigational New Drug (IND) Application for UX016, a Sialic Acid Prodrug for the Treatment of GNE Myopathy

Mereo BioPharma Reports Full Year 2025 Financial Results and Provides Corporate Highlights

Ultragenyx Announces Positive 36-Week Data from Phase 3 Study of DTX301 AAV8 Gene Therapy for the Treatment of Ornithine Transcarbamylase (OTC) Deficiency

Ultragenyx Announces U.S. FDA Acceptance and Priority Review of the Biologics License Application (BLA) for DTX401 AAV Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa)

Ultragenyx Reports Fourth Quarter and Full Year 2025 Financial Results and Corporate Update

Ultragenyx to Host Conference Call for Fourth Quarter and Full-Year 2025 Financial Results and Corporate Update

Ultragenyx Announces Positive Longer-Term Data Demonstrating Treatment with UX111 Gene Therapy Results in Sustained, Significant Reductions in CSF-HS and Continued Meaningful Improvements in Clinical Function Across Multiple Developmental Domains in Children with Sanfilippo Syndrome (MPS IIIA)

Ultragenyx Announces Positive Longer-Term Data Demonstrating Treatment with UX111 Gene Therapy Results in Sustained, Significant Reductions in CSF-HS and Continued Meaningful Improvements in Clinical Function Across Multiple Developmental Domains in C...

Ultragenyx Resubmits Biologics License Application for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA) to U.S. FDA

Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)

Ultragenyx Provides Financial and Business Updates at J.P. Morgan Annual Healthcare Conference

Ultragenyx to Present at the 44th Annual J.P. Morgan Healthcare Conference

Ultragenyx Completes Rolling Submission of Biologics License Application (BLA) to U.S. FDA for DTX401 AAV Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa)

Ultragenyx Announces Phase 3 Orbit and Cosmic Results for Setrusumab (UX143) in Osteogenesis Imperfecta

Tactical Resources Announces Share Consolidation

Ultragenyx to Participate in Investor Conferences in December

Ultragenyx Reports Third Quarter 2025 Financial Results and Corporate Update

Ultragenyx Announces Sale of a Portion of Future North American Royalties on Crysvita® (burosumab) for $400 Million to OMERS Life Sciences

Ultragenyx Announces First Patient Dosed in Aurora Study Evaluating GTX-102 in Additional Angelman Syndrome Genotypes and Age Groups

Ultragenyx to Host Conference Call for Third Quarter 2025 Financial Results and Corporate Update

Ultragenyx Appoints Eric Olson as Chief Business Officer and Executive Vice President

Ultragenyx Announces Positive Longer-term Data from Phase 3 Study of DTX401 AAV Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia (GSDIa)

Ultragenyx to Participate at Investor Conferences in September

Rezolute Appoints Seasoned Commercial Executive Sunil Karnawat as Chief Commercial Officer

Ultragenyx Initiates Rolling Submission of Biologics License Application (BLA) to U.S. FDA for DTX401 AAV Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia (GSDIa)

Ultragenyx Reports Second Quarter 2025 Financial Results and Corporate Update

Ultragenyx Completes Enrollment of Phase 3 Aspire Study Evaluating GTX-102 for the Treatment of Angelman Syndrome

Ultragenyx to Host Conference Call for Second Quarter 2025 Financial Results and Corporate Update

New Pedestrian Access from Inner Belt to East Somerville MBTA Station Now Open to the Public

Health Canada Extends the Approval of Evkeeza® (evinacumab) to Children as Young as 6-months Old with Homozygous Familial Hypercholesterolemia (HoFH)

Ultragenyx Receives Complete Response Letter from FDA for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)

Ultragenyx and Mereo BioPharma Announce UX143 Phase 3 Orbit Study for Osteogenesis Imperfecta Progressing to Final Analysis

Ultragenyx Receives Breakthrough Therapy Designation for GTX-102 in Angelman Syndrome