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 IIIA (MPS IIIA) (S31.001)
Describe safety and efficacy of a single administration of UX111 in children with MPS IIIA
Authors
- Heather Lau (ORCID: https://orcid.org/0000-0003-1690-9457)
- Maria Fuller (ORCID: https://orcid.org/0000-0001-9092-8942)
- Kristen Truxal (ORCID: https://orcid.org/0000-0002-8787-9411)
- Lucía Dougherty‐de Miguel (ORCID: https://orcid.org/0000-0002-8865-2438)
- Kevin M. Flanigan (ORCID: https://orcid.org/0000-0001-6440-3376)
- Nicholas Smith (ORCID: https://orcid.org/0000-0003-2409-9239)
- María Luz Couce (ORCID: https://orcid.org/0000-0003-4861-9905)
- Deepa Rajan (ORCID: https://orcid.org/0000-0001-5699-8541)
- Mireia del Toro (ORCID: https://orcid.org/0000-0002-8163-1853)
- Melissa M. Wolf (ORCID: https://orcid.org/0000-0003-2038-6978)
- Kaushik Patra
- María J. de Castro
- E. Monteagudo
Institutions
- South Australia Pathology (AU)
- Nationwide Children's Hospital (US)
- University of Pittsburgh (US)
- Complejo Hospitalario Universitario de Santiago (ES)
- Ultragenyx Pharmaceutical (United States) (US)
- Vall d'Hebron Hospital Universitari (ES)
- Children's Hospital of Pittsburgh (US)
- Instituto de Investigación Sanitaria de Santiago (ES)
- The University of Adelaide (AU)
Publication Details
- Journal
- Neurology
- Published
- 2025-04-07
- DOI
- https://doi.org/10.1212/wnl.0000000000211655
- Citations
- 2
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 2.33