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 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 ...
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, ...
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 ...
Sanfilippo syndrome, also known as mucopolysaccharidosis type III (MPS III), is considered an orphan disease, which classifies it for special considerations in drug development and policy. It is a ...
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 ...
Runners lace up to raise money, awareness as Sachse 10-year-old battles rare, fatal genetic syndrome
There are still moments when life feels normal inside of the Sink home. But for John and Katharine Sink, not a day goes by that they don’t think of the diagnosis doctors gave 10-year-old Declan six ...
Ultragenyx announced significant cognitive improvements in Sanfilippo syndrome type A patients treated with UX111 gene therapy, presented at WORLDSymposium™ 2025. Ultragenyx Pharmaceutical Inc.
KNOXVILLE, Tenn. — Bryan and Blair Begbie said all they want for Rowan — their youngest daughter — is the chance to be a kid. However, the Food and Drug Administration issued what is called a Complete ...
Torrance, California, June 21, 2024 (GLOBE NEWSWIRE) -- As a neurodegenerative disease characterized by childhood onset dementia, Sanfilippo syndrome causes immense suffering in many ways, including ...
KNOXVILLE, Tenn. — Bryan and Blair Begbie said all they want for Rowan — their youngest daughter — is the chance to be a kid. However, the Food and Drug Administration issued what is called a Complete ...
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