Real-world clinical outcomes of patients with CLN2 disease treated with cerliponase alfa.
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| Title: | Real-world clinical outcomes of patients with CLN2 disease treated with cerliponase alfa. |
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| Authors: | Schulz A; Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.; German Center for Child and Adolescent Health (DZKJ) Hamburg Partner Site, Hamburg, Germany., Schwering C; Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany., Wibbeler E; Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany., Westermann LM; Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany., Hagenah L; Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany., Lezius S; Institute of Medical Biometry and Epidemiology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany., Jha A; BioMarin Pharmaceutical Inc., Novato, CA, United States., Hunt A; BioMarin Pharmaceutical Inc., Novato, CA, United States., Slasor P; BioMarin Pharmaceutical Inc., Novato, CA, United States., Reisewitz P; BioMarin Pharmaceutical Inc., Novato, CA, United States., Nickel M; Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany. |
| Source: | Frontiers in neurology [Front Neurol] 2025 Mar 14; Vol. 16, pp. 1516026. Date of Electronic Publication: 2025 Mar 14 (Print Publication: 2025). |
| Publication Type: | Journal Article |
| Journal Info: | Publisher: Frontiers Research Foundation Country of Publication: Switzerland NLM ID: 101546899 Publication Model: eCollection Cited Medium: Print ISSN: 1664-2295 (Print) Linking ISSN: 16642295 NLM ISO Abbreviation: Front Neurol Subsets: PubMed not MEDLINE |
| Database: | MEDLINE Ultimate |
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| ISSN: | 1664-2295 |
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| DOI: | 10.3389/fneur.2025.1516026 |
