Real-world clinical outcomes of patients with CLN2 disease treated with cerliponase alfa
Cerliponase alfa for real-world CLN2 disease treatment slowed decline in motor and language function and demonstrated an acceptable safety profile.
Authors
- Christoph Schwering (ORCID: https://orcid.org/0000-0002-3716-1575)
- Miriam Nickel (ORCID: https://orcid.org/0000-0002-1870-1142)
- Lena Marie Westermann
- Pascal Reisewitz
- Eva Wibbeler (ORCID: https://orcid.org/0000-0001-7451-3616)
- Abigail Hunt
- Peter Slasor
- Susanne Lezius (ORCID: https://orcid.org/0000-0002-4832-4333)
- Luca Hagenah
- Ashok Kumar Jha (ORCID: https://orcid.org/0000-0003-4100-3286)
- Angela Schulz (ORCID: https://orcid.org/0000-0001-9156-4861)
Institutions
- Universität Hamburg (DE)
- University Medical Center Hamburg-Eppendorf (DE)
- BioMarin (United States) (US)
Publication Details
- Journal
- Frontiers in Neurology
- Published
- 2025-03-14
- DOI
- https://doi.org/10.3389/fneur.2025.1516026
- Citations
- 8
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 9.31