Rally for Rowan: Family fights for awareness and research after daughter’s rare diagnosis
The Genetic and Rare Diseases Information Center estimates that fewer than 5,000 people in the U.S. have it. The syndrome is ...
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RARE Reports New Upbeat Data From Sanfilippo Syndrome Drug Study
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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This Family Advocates For Rare Sanfilippo Syndrome In Hopes That Others Have Better Chances
Soon after, Logan was diagnosed with Sanfilippo syndrome. As Logan’s mom, Noelle, described: “Sanfilippo syndrome is a ...
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Ultragenyx Announces New Data For UX111 Gene Therapy In Treating Sanfilippo Syndrome Type A
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 ...
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FDA Grants Priority Review to Ultragenyx's BLA for Sanfilippo Syndrome Type A Gene Therapy
The firm is seeking accelerated approval for UX111 using reductions in levels of heparan sulfate in cerebrospinal fluid as a surrogate endpoint.
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Ultragenyx Submits Biologics License Application to the U.S. FDA for UX111 AAV Gene Therapy for the Treatment of Sanfilippo Syndrome Type A (MPS IIIA)
The BLA submission for UX111 is supported by available data, including from the ongoing pivotal Transpher A study, demonstrating treatment with UX111 resulted in rapid and sustained decreased levels ...
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Rally for Rowan: Family fights for awareness and research after daughter’s rare diagnosis
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.