Latest News and Press Releases

Want to stay updated on the latest news?

Sign into Reader Account Create Reader Account

February 06, 2024 08:00 ET | Source: Ultragenyx Pharmaceutical Inc.

Ultragenyx Announces Data Demonstrating Treatment with UX111 Results in Significant Reduction in Heparan Sulfate Exposure in Cerebrospinal Fluid Correlated with Improved Long-term Cognitive Function in Patients with Sanfilippo Syndrome Type A (MPS IIIA)

NOVATO, Calif., Feb. 06, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced data demonstrating treatment with UX111 (ABO-102) AAV gene therapy resulted in rapid...
February 05, 2024 16:05 ET | Source: Ultragenyx Pharmaceutical Inc.

Ultragenyx Receives PRIME Designation from European Medicines Agency (EMA) for GTX-102 for the Treatment of Angelman Syndrome

GTX-102 is the first Angelman syndrome therapeutic candidate to receive PRIME designation Phase 1/2 study fully enrolled; expansion data expected in first half of 2024 NOVATO, Calif., Feb. 05, 2024...
January 31, 2024 08:00 ET | Source: Ultragenyx Pharmaceutical Inc.

Ultragenyx anuncia la aprobación de Dojolvi® (triheptanoína) para su inclusión en el Compendio Nacional de Insumos para la Salud de México en el tratamiento de los trastornos de oxidación de ácidos grasos de cadena larga (LC-FAOD)

CIUDAD DE MÉXICO, Jan. 31, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc, compañía biofarmacéutica dedicada al desarrollo y comercialización de tratamientos novedosos para enfermedades...
January 25, 2024 16:05 ET | Source: Ultragenyx Pharmaceutical Inc.

Ultragenyx Announces Completion of Dosing Across Stage 1 Cohorts in Pivotal Phase 1/2/3 Cyprus2+ Study Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease

Safety and initial efficacy data from Stage 1 expected in the first half of 2024 Dose selection and initiation of Stage 2 to follow in the second half of 2024 NOVATO, Calif., Jan. 25, 2024 (GLOBE...
January 19, 2024 16:00 ET | Source: Ultragenyx Pharmaceutical Inc.

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

NOVATO, Calif., Jan. 19, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for...
January 07, 2024 11:00 ET | Source: Ultragenyx Pharmaceutical Inc.

Ultragenyx Reports Preliminary 2023 Revenue; Guidance for 2024 Revenue and Cash Usage; Pipeline Updates and 2024 Milestones

Preliminary 2023 Total Revenue of $430 million to $435 million, Crysvita® Revenue of $325 million to $330 million and Dojolvi® revenue of $70 million to $71 million 2024 expected Total Revenue...
January 04, 2024 08:00 ET | Source: Ultragenyx Pharmaceutical Inc.

Ultragenyx Receives Positive Recommendation from NICE in the U.K. for Evkeeza® ▼ (evinacumab) for Adolescents and Adults Aged 12 Years and Older with Homozygous Familial Hypercholesterolemia (HoFH)

NOVATO, Calif., Jan. 04, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the National Institute for Health and Care Excellence (NICE) has issued a final...
January 03, 2024 08:00 ET | Source: Ultragenyx Pharmaceutical Inc.

Ultragenyx Announces Completion of Enrollment in Global Phase 1/2 Trial of GTX-102 in Pediatric Patients with Angelman Syndrome

NOVATO, Calif., Jan. 03, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for...
January 02, 2024 16:05 ET | Source: Ultragenyx Pharmaceutical Inc.

Ultragenyx to Present at the 42nd Annual J.P. Morgan Healthcare Conference

NOVATO, Calif., Jan. 02, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for...
December 21, 2023 16:30 ET | Source: Ultragenyx Pharmaceutical Inc.

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

NOVATO, Calif., Dec. 21, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for...