Discover Sanfilippo Syndrome a rare genetic disorder affecting childrens development Learn about its symptoms treatment ...

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

Ultragenyx Pharmaceutical is a mid-cap biotech firm with a promising pipeline and an optimistic outlook from analysts. Click ...

Soon after, Logan was diagnosed with Sanfilippo syndrome. As Logan’s mom, Noelle, described: “Sanfilippo syndrome is a ...

GREENVILLE, S.C. (FOX Carolina) - Clemson researchers believe they’re on the verge of a breakthrough treatment for Sanfilippo Syndrome, which is also known as childhood Alzheimer’s.

Denali Therapeutics DNLI has performed well in the past 12 months amid a volatile market. Shares of the company have gained ...

Sanfilippo Syndrome Type A, or MPS IIIA, is a rare, fatal neurodegenerative disease in which the body is unable to break down a complex sugar molecule called heparan sulfate. This disorder ...

Denali Therapeutics showcases its BBB platform at J.P. Morgan Conference, highlighting DNL310's Hunter syndrome progress and plans for commercialization.

such as DNL126 for Sanfilippo syndrome type A. Most recently, Denali Therapeutics initiated dosing in a global BEACON Phase 2a trial of BIIB122 (DNL151) for LRRK2-associated Parkinson’s disease.

The BLA submission for UX111 is supported by available data, including from the ongoing pivotal Transpher A study, demonstrating treatment with UX111 resulted in rapid and sustained decreased levels ...

“In 2025, we will continue to expand our commercial base of business while we also prepare for the potential launch of our first gene therapy, in Sanfilippo syndrome, and to file a BLA for our ...

This past December, Clark was diagnosed with Sanfilippo Syndrome. A neurological disease that causes Alzheimer-like symptoms in children. Clark’s parents say this surprise means everything to ...