Ultragenyx Announces FDA Acceptance and Priority Review of the Biologics License Application (BLA) for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA). Retrieved February 19, 2025. 注:以上资讯来源于网络,由香港济民药业整理编辑(如有错漏,请帮忙指正),只为提供全球最新上市药品的...
(RTTNews) - Ultragenyx Pharmaceutical Inc. (RARE), announced on Thursday that it has submitted a Biologics License Application to the U.S. FDA for UX111, an AAV gene therapy for Sanfilippo Syndrome Type A.
Treatment with UX111 gene therapy rapidly reduced heparan sulfate (HS) exposure in cerebrospinal fluid (CSF) and improved long-term cognitive function in children with mucopolysaccharidosis type IIIA (MPS IIIA)Heather A. LauKaushik PatraMelissa Wolf...
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[1] Ultragenyx Announces New Data Demonstrating that Treatment with UX111 AAV Gene Therapy Significantly Improved Clinical Function Across Multiple Developmental Domains in Children with Sanfilippo Syndrome Type A (MPS IIIA) Correlated with Sustained Reductions in CSF-HS,Feb 05, 2025. ...
4、Genentech官网、https://www.gene.com/media/press-releases; 5、临床试验的临床结果,https://clinicaltrials.gov/; 6、IMpassion130: updated overall survival (OS) from a global, randomized, double-blind, placebo-controlled, Phase III study of atezolizumab...
Interim results of Transpher A, a multicenter, single-dose clinical trial of UX111 gene therapy for Sanfilippo syndrome type A (mucopolysaccharidosis IIIA)doi:10.1016/j.ymgme.2022.107101K. FlaniganN. SmithM. CouceD. RajanK. TruxalKimL. McBride...