Soon after, Logan was diagnosed with Sanfilippo syndrome. As Logan’s mom, Noelle, described: “Sanfilippo syndrome is a ...

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).

Sanfilippo syndrome type A is a rare, fatal condition with no approved treatment that affects approximately 3,000 to 5,000 ...

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.

(RARE), a biopharmaceutical company, on Wednesday reported encouraging data from its ongoing clinical program of UX111, a gene therapy for Sanfilippo Syndrome Type A or MPS IIIA, a rare ...

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 ...

Dr. Brennan’s current areas of research focus on speech, language, and communication in individuals with rare conditions, including Smith-Magenis Syndrome (SMS), Sanfilippo Syndrome, Autoimmune ...