One such condition is Sanfilippo syndrome, which creates significant hurdles for affected children and their families. This condition not only impacts physical development but also cognitive and ...

As Logan’s mom, Noelle, described: “Sanfilippo syndrome is a terminal, neurodegenerative rare disease. Children lose all the ...

Mackay says children with Sanfilippo syndrome have too much build-up of heparan sulfate in the lysosome of a cell. This build-up causes damage to the brain. “We know the cause, but what we don ...

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

(RTTNews) - Ultragenyx Pharmaceutical Inc (RARE) today announced that the FDA has accepted for priority review its Biologics License Application for UX11, an AAV gene therapy proposed for the ...

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.

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

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 ...

Dr. Brennan’s current areas of research focus on speech, language, and communication in individuals with rare conditions, including Smith-Magenis Syndrome (SMS), Sanfilippo Syndrome ... in school-age ...

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).

The firm is seeking accelerated approval for UX111 using reductions in levels of heparan sulfate in cerebrospinal fluid as a surrogate endpoint.