Long-term data demonstrate that robust reductions and normalization in key biomarkers from baseline were maintained over time with continued improvement in hearing, cognition and adaptive behaviorLong ...

After winning a breakthrough therapy designation for its Hunter syndrome enzyme replacement therapy, Denali Therapeutics is ...

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 ...

Mr. Cleary previously served as Senior Vice President ("SVP”) of Corporate Development at Medtronic plc (NYSE: MDT) ("Medtronic”), where he played a key role in establishing the strategic ...

The Grand Rapids Police Department will face the Kentwood Police Department in just one of the matchups scheduled for the ...

Tierney LaSotta was diagnosed with Sanfilippo Syndrome, a rare condition also known as Childhood Dementia because of its ...

What is Zollinger-Ellison syndrome? Zollinger-Ellison syndrome (ZES) is a rare digestive disorder. If you have ZES, you likely have 1 or more tumors in the first part of the small intestine, the ...

For some families, this is a daily reality due to rare genetic disorders. One such condition is Sanfilippo syndrome, which creates significant hurdles for affected children and their families.

Gallerie d’Italia, Intesa Sanpaolo’s museum in Milan, is now presenting Una collezione inattesa. Viaggio nel contemporaneo tra pittura e scultura [An unexpected collection. ITALICS is the first ...

17, 2025, in Arlington, Texas, with wife Ashyln, son Brooks and daughter Penelope while holding the No. 21 jersey that has special significance after his daughter was born with Down syndrome. (AP ...