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This Family Advocates For Rare Sanfilippo Syndrome In Hopes That Others Have Better ChancesSoon after, Logan was diagnosed with Sanfilippo syndrome. As Logan’s mom, Noelle, described: “Sanfilippo syndrome is a ...
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College football fans unite to raise thousands for young Ohio State fan with rare diseaseIt was revealed that Clark had Sanfilippo syndrome, a rare disease that is often described as childhood Alzheimers. "In my mind, I'm sitting there thinking, like, I'm not going to watch him grow ...
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).
Dr. Eric Crombez, Chief Medical Officer at Ultragenyx, stated, "The data support UX111's potential to provide meaningful benefits to children with Sanfilippo Syndrome, especially for those in ...
The Novato-based maker of biopharmaceutical treatments for rare diseases plans to release this year a new gene therapy for Sanfilippo syndrome, a rare metabolism disorder. Ultragenyx ...
The company expects to launch a new gene therapy for Sanfilippo syndrome, a rare metabolism disorder, this year. The company also reported a reduced net loss in 2024 of $569.83 million ...
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