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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 ...
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.
Nevertheless, the new disappointment with DNL343 is another major disappointment to ALS patients, who have limited drug ...
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 ...
Ultragenyx Pharmaceutical Inc. (Nasdaq: RARE) announced fourth-quarter results Jan. 12 of $164.87 million. That’s a 29% ...
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).
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