Jack Baird, who is four years old and from Sunderland, has the condition Sanfilippo Disease, also known as Mucopolysaccharidosis (MPS) III, which affects around one in 85,000 people in the UK.

Noelle also commented on her role as a parent, advocating for awareness of Sanfilippo syndrome on social media. “The main ...

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.

Clemson researchers believe they’re on the verge of a breakthrough treatment for Sanfilippo Syndrome, which is also known as childhood Alzheimer’s. There is no cure and most children pass away when ...

RARE and its partner, Mereo BioPharma, are jointly developing UX143 (setrusumab) monoclonal antibody forpediatric and young adult patients with osteogenesis imperfecta (OI) in two late-stage ...

(NASDAQ: RARE) today announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA or the Agency) seeking accelerated approval for UX111 (ABO-102) AAV ...

(RARE), announced on Thursday that it has submitted a Biologics License Application to the U.S. FDA for UX111, an AAV gene therapy for Sanfilippo Syndrome Type A. Sanfilippo Syndrome Type A ...

As a dual diagnosis therapist, I specialize in trauma and addiction treatment. I have experience working with adults, children, and adolescents. All the therapeutic modalities I work in are trauma ...

“This may be surprising to people, 70% of human disease genes have fruit fly counterparts,” Mackay said. Mackay says children with Sanfilippo syndrome have too much build-up of heparan sulfate ...