Reports Q4 revenue $164.88M, consensus $158.71M. “We have created a next-generation rare disease company on a pathway to profitability with ...

Total Revenue of $560 million, exceeding guidanceCrysvita® revenue of $410 million and Dojolvi® revenue of $88 million2025 Financial ...

Glycogen Storage Disease (GSD): A group of inherited disorders caused by enzyme deficiencies that affect the metabolism of glycogen. Neutropenia: A condition characterized by an abnormally low ...

While the metabolic defects affect all cells, clinical organ involvement usually occurs only in the presence of substrate excess or metabolic pathway activation. Cardiac disease is particularly ...

Though she acknowledges that there’s ‘still a lot of research to be done’, her efforts to decipher the findings of existing ...

Good afternoon, and welcome to the Ultragenyx Fourth Quarter and Full Year 2024 Financial Results Conference Call. At this time, all participants are in a listen-only-mode. At the end of the prepared ...

Ultragenyx Pharmaceutical RARE reported fourth-quarter 2024 loss of $1.39 per share, wider than the Zacks Consensus Estimate ...

These children may not have overt signs of an underlying muscle disease (such as fixed weakness or myalgia ... have autosomal recessive inheritance. Glycogenoses, or glycogen storage disorders, are ...

Our findings contribute to a better understanding of the mechanisms linking cellular senescence and fatty liver disease and support ... profound changes in hepatocyte metabolism, with increased lipid ...

Ultragenyx Pharmaceutical RARE reported new findings from an ongoing, pivotal study of its investigational candidate, UX111 (ABO-102) AAV gene therapy, for Sanfilippo syndrome type A (MPS IIIA). Per ...

This poses a problem to patients with McArdle disease when there is a need for anaerobic metabolism and a high glycolytic ... like to thank Mr Andrew Wakelin of the Association for Glycogen Storage ...