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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.
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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  Data: Real-world clinical outcomes of patients with CLN2 disease treated with cerliponase alfa.
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  Data: <searchLink fieldCode="AU" term="%22Schulz+A%22">Schulz A</searchLink>; Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.; German Center for Child and Adolescent Health (DZKJ) Hamburg Partner Site, Hamburg, Germany.<br /><searchLink fieldCode="AU" term="%22Schwering+C%22">Schwering C</searchLink>; Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.<br /><searchLink fieldCode="AU" term="%22Wibbeler+E%22">Wibbeler E</searchLink>; Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.<br /><searchLink fieldCode="AU" term="%22Westermann+LM%22">Westermann LM</searchLink>; Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.<br /><searchLink fieldCode="AU" term="%22Hagenah+L%22">Hagenah L</searchLink>; Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.<br /><searchLink fieldCode="AU" term="%22Lezius+S%22">Lezius S</searchLink>; Institute of Medical Biometry and Epidemiology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.<br /><searchLink fieldCode="AU" term="%22Jha+A%22">Jha A</searchLink>; BioMarin Pharmaceutical Inc., Novato, CA, United States.<br /><searchLink fieldCode="AU" term="%22Hunt+A%22">Hunt A</searchLink>; BioMarin Pharmaceutical Inc., Novato, CA, United States.<br /><searchLink fieldCode="AU" term="%22Slasor+P%22">Slasor P</searchLink>; BioMarin Pharmaceutical Inc., Novato, CA, United States.<br /><searchLink fieldCode="AU" term="%22Reisewitz+P%22">Reisewitz P</searchLink>; BioMarin Pharmaceutical Inc., Novato, CA, United States.<br /><searchLink fieldCode="AU" term="%22Nickel+M%22">Nickel M</searchLink>; Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
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  Data: <searchLink fieldCode="JN" term="%22101546899%22">Frontiers in neurology</searchLink> [Front Neurol] 2025 Mar 14; Vol. 16, pp. 1516026. <i>Date of Electronic Publication: </i>2025 Mar 14 (<i>Print Publication: </i>2025).
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Frontiers+Research+Foundation%22">Frontiers Research Foundation </searchLink><i>Country of Publication: </i>Switzerland <i>NLM ID: </i>101546899 <i>Publication Model: </i>eCollection <i>Cited Medium: </i>Print <i>ISSN: </i>1664-2295 (Print) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2216642295%22">16642295 </searchLink><i>NLM ISO Abbreviation: </i>Front Neurol <i>Subsets: </i>PubMed not MEDLINE
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              Text: 2025 Mar 14
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