The mucopolysaccharidosis market is witnessing a surge in innovation, driven by cutting-edge gene therapies, enzyme replacement treatments, and novel drug developments. The market is poised for ...
Total Revenue of $560 million, exceeding guidanceCrysvita® revenue of $410 million and Dojolvi® revenue of $88 million2025 Financial ...
Reports Q4 revenue $164.88M, consensus $158.71M. “We have created a next-generation rare disease company on a pathway to profitability with ...
The child tax credit provides big benefits for families with children, but you may have to wait a bit longer for your tax refund. Thomas is a native of upstate New York and a graduate of the ...
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).
An Orange couple who lost their son to a rare genetic condition are determined to continue raising awareness to improve the lives of diagnosed children and their families.
Notably, older children or those with more advanced disease retained ... received several designations from the FDA that could expedite its review process. Sanfilippo syndrome type A is a rare, fatal ...
Voters are even wondering about her marital status and whether or not she has any children. In his announcement, Trump called Bondi a “smart and tough” person, whom he’s known for “many ...
Dr. Eric Crombez, Chief Medical Officer at Ultragenyx, stated, "The data support UX111's potential to provide meaningful benefits to children with Sanfilippo Syndrome, especially for those in ...
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