An Intravenous Brain-Penetrant Enzyme Therapy for Mucopolysaccharidosis II
In participants with MPS II, tividenofusp alfa treatment was commonly associated with adverse events. Heparan sulfate, the primary substrate that accumulates in the CSF and urine in persons with MPS II, appeared to decrease to levels within the range of unaffected children. A randomized trial is ongoing to further evaluate these effects. (Funded by Denali Therapeutics; ClinicalTrials.gov number, NCT04251026; EudraCT number, 2019-004909-27.).
Authors
- Carole Ho (ORCID: https://orcid.org/0000-0003-3623-8267)
- Joseph A. Muenzer
- Akhil Bhalla
- Simon A. Jones (ORCID: https://orcid.org/0000-0001-7297-9711)
- Natalie Engmann
- Matthew D. Troyer (ORCID: https://orcid.org/0000-0002-0821-7509)
- Fabian Model (ORCID: https://orcid.org/0009-0002-6319-9902)
- Imanol Zubizarreta
- Deepa Rajan (ORCID: https://orcid.org/0000-0001-5699-8541)
- Ryan J. Watts (ORCID: https://orcid.org/0000-0002-4973-5089)
- Johanna M.P. van den Hout (ORCID: https://orcid.org/0000-0001-5150-0767)
- Paul R. Harmatz
- Barbara K. Burton
- Peter Chin
- Wei Dong
- John J. Mitchell
Institutions
- University of North Carolina at Chapel Hill (US)
- Lurie Children's Hospital (US)
- Montreal Children's Hospital (CA)
- McGill University Health Centre (CA)
- Erasmus MC (NL)
- Manchester University NHS Foundation Trust (GB)
- Denali Therapeutics (United States) (US)
- Children's Hospital of Pittsburgh (US)
- UCSF Benioff Children's Hospital (US)
Publication Details
- Journal
- New England Journal of Medicine
- Published
- 2025-12-26
- DOI
- https://doi.org/10.1056/nejmoa2508681
- Citations
- 4
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 4.66