Sanfilippo syndrome is a fatal, childhood, neurodegenerative, disorder—with no approved treatment—that involves neuroinflammation among multiple pathologies. Characterized by childhood onset dementia, ...

As a neurodegenerative disease characterized by childhood onset dementia, Sanfilippo syndrome causes immense suffering in many ways, including pain, loss of speech, extreme agitation, and distress, ...

Add Yahoo as a preferred source to see more of our stories on Google. As a pediatrician, I’ve dedicated my career to advocating for children’s health. But when my daughter, Eliza, was diagnosed with ...

NATCHITOCHES, La. -- On April 4, a mother and father received heartbreaking news that would alter the course of their lives forever. Their daughter, Lydia, just 18 months old, was diagnosed with a ...

Analysis from continued follow-up of Phase 1/2 clinical trial data in Hunter syndrome (MPS II) reinforces potential for tividenofusp alfa ...

Not everyone is given equal opportunities in life, but even when faced with the most difficult challenges, it’s not the circumstances that define us, but our outlook. Logan was born in 2007 alongside ...

Ultragenyx announced significant cognitive improvements in Sanfilippo syndrome type A patients treated with UX111 gene therapy, presented at WORLDSymposium™ 2025. Ultragenyx Pharmaceutical Inc.

NEW ORLEANS — It is said, "A mother's love is a force that can move mountains." It was a year ago that we brought you the story of a mother desperately searching for a cure for her daughter's rare, ...

As a pediatrician, I’ve dedicated my career to advocating for children’s health. But when my daughter, Eliza, was diagnosed with Sanfilippo syndrome, a fatal childhood neurological disease, I realized ...