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Ultragenyx Releases 2025 Impact Report Emphasizing Commitment to Rare Disease Patients, Innovation, and Global Impact
Ultragenyx Pharmaceutical Inc. released its 2025 Impact Report, detailing the company's work on behalf of patients and families living with rare and ultra-rare diseases. In 2025, Ultragenyx advanced five investigational therapies in pivotal clinical programs with the potential to reach tens of thousands of patients around the world — while continuing to build on years of work to support patients who cannot yet access approved treatments, which have now reached individuals in 50 countries.“The rare disease
Ultragenyx Announces Positive Data from Study of Gene Therapy Treatment for OTC Deficiency
Today Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced positive results from its Phase 3 Enh3ance study of DTX301, an investigational AAV8 gene therapy for the treatment of ornithine transcarbamylase (OTC) deficiency. At Week 36 in the randomized, double-blind placebo-controlled period of the trial, patients demonstrated a statistically significant and clinically meaningful 18% reduction in 24-hour plasma ammonia (AUC0-24) compared to placebo, and maintained average ammonia AUC0-24 in the normal
BACC Newsletter - July 2025
Member News and Information Lieutenant Governor Driscoll Joins BACC for Life Sciences Panel The BACC, in collaboration with Massachusetts State Representative Ken Gordon, sponsored a Life Science Panel with Lt. Governor Kim Driscoll to discuss the impact of NIH funding cuts to the local life science ecosystem see more... Lahey Hospital & Medical Center Celebrates Opening of New Clinical Research Center LHMC marked a major milestone with the official opening of its new Clinical Research Center,
Lieutenant Governor Kim Driscoll Joins BACC for Life Sciences Panel
The Burlington Area Chamber of Commerce (BACC), in collaboration with Massachusetts State Representative Ken Gordon, sponsored a Life Science Panel with Lieutenant Governor Kim Driscoll to discuss the impact of the National Institutes of Health (NIH) funding cuts to the local life science ecosystem. The event was held at Northeastern University’s Innovation Campus at Burlington, and included a select audience of area business leaders, life science professionals, and local municipal officials. Photo
