Diagnosed at a young age with Sanfilippo syndrome, a rare genetic disorder resembling childhood Alzheimer’s disease, Joseph ...

The progressive brain disorder, which causes children to lose speech, mobility and memory, has no cure and families like Sara ...

She and a group of local women believed Rowan had MPS 3A, more commonly known as Sanfilippo syndrome. A few weeks later, a geneticist confirmed the devastating news: Rowan had Sanfilippo.

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).

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.

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.

AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA) under the accelerated approval pathway. Earlier this year, the FDA aligned with Ultragenyx, after a thorough ...

the couple learned from a neurologist that Declan is one of the one-in-70,000 kids born in the U.S. with Sanfilippo syndrome. Get top local stories delivered to you every morning with NBC DFW's ...

Sanfilippo syndrome, also known as mucopolysaccharidosis type III, is a rare life-threatening disorder that interferes with metabolism. While it doesn’t have a cure, some symptoms can be treated ...

Soon after, Logan was diagnosed with Sanfilippo syndrome. As Logan’s mom, Noelle, described: “Sanfilippo syndrome is a terminal, neurodegenerative rare disease. Children lose all the skills ...

The diagnosis turned out to be more challenging: Emmett Doucette had developed a rare condition called Sanfilippo syndrome. “We’re just, typical family that had this diagnosis,” McCabe says ...