Analysis of Potential Biomarkers and Clinical Scale in Atypical CLN2 Enzyme Replacement Therapy Patients (P10-6.010)
To evaluate potential biomarkers of disease progression in patients with atypical Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) disease currently undergoing enzyme replacement therapy (ERT) with a recombinant human tripeptidyl peptidase 1 (TPP1) protein.
Authors
- Raymond Wang (ORCID: https://orcid.org/0000-0001-6494-7613)
- Joffre Olaya (ORCID: https://orcid.org/0000-0002-2039-6490)
- Gianna Fote (ORCID: https://orcid.org/0000-0002-1854-7894)
- Chloe E. Villars
- Victoria C. Stewart
Institutions
- Children's Hospital of Orange County (US)
- University of California, Irvine (US)
- Neurological Surgery (US)
Publication Details
- Journal
- Neurology
- Published
- 2025-04-07
- DOI
- https://doi.org/10.1212/wnl.0000000000210983
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00