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 ...

(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 ...

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 ...

Emil Kakkis; President, Chief Executive Officer, Director; Ultragenyx Pharmaceutical Inc Erik Harris; Executive Vice President, Chief Commercial Officer; Ultragenyx Pharmaceutical Inc Howard Horn; ...

Notably, older children or those with more advanced ... from the FDA that could expedite its review process. Sanfilippo syndrome type A is a rare, fatal condition with no approved treatment ...

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 ...