Insights into immunogenicity and therapeutic strategies to mitigate the immune response in infantile-onset Pompe disease: a comprehensive systematic literature review
The study concluded that CRIM status determination is important in patients with IOPD and impacts management approaches. ITI use has been predominantly reported in CRIM-negative patients to improve the clinical outcomes, with other important factors being early initiation of ERT and treatment above label dose of alglucosidase alpha and many are doing upto 40 mg/kg/2 weeks.
Authors
- Colin D. Glen
- Jennifer Heuterman
- Nadia Daba
- Susan Sparks (ORCID: https://orcid.org/0000-0002-7469-6691)
- Johanna M. P. van den Hout (ORCID: https://orcid.org/0000-0001-8091-263X)
- Yin-Hsiu Chien
- David Kronn
- Priya S. Kishnani
- Mei Han
- Andreas Hahn
Institutions
- New York Medical College (US)
- Justus-Liebig-Universität Gießen (DE)
- Erasmus MC (NL)
- AVEO Oncology (United States) (US)
- Sanofi (Netherlands) (NL)
- Lucas Research (US)
- Duke Medical Center (US)
- National Taiwan University Hospital (TW)
- Erasmus University Rotterdam (NL)
Publication Details
- Journal
- Frontiers in Immunology
- Published
- 2026-01-08
- DOI
- https://doi.org/10.3389/fimmu.2025.1690312
- Citations
- 2
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 65.68
Funders
- Sanofi