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

The daughter of a Kent County sheriff’s deputy who is fighting a rare genetic disorder will benefit from this year’s Robert ...

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

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

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

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.

A study into a new treatment for Sanfilippo disease, a rare and fatal condition which causes progressive dementia in children, is progressing well with results set to be published later this year.

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 ...

Funded by charity, The Society for Mucopolysaccharide Diseases (The MPS Society)*, the study could alter the lives of patients with Sanfilippo Disease – a fatal inherited condition which causes ...

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.

“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 ...