Safety and efficacy of cerliponase alfa in children with neuronal ceroid lipofuscinosis type 2 (CLN2...
Safety and efficacy of cerliponase alfa in children with neuronal ceroid lipofuscinosis type 2 (CLN2 disease): an open-label extension study
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Author / Creator
Schulz, Angela , Specchio, Nicola , de los Reyes, Emily , Gissen, Paul , Nickel, Miriam , Trivisano, Marina , Aylward, Shawn C , Chakrapani, Anupam , Schwering, Christoph , Wibbeler, Eva , Westermann, Lena Marie , Ballon, Douglas J , Dyke, Jonathan P , Cherukuri, Anu , Bondade, Shailesh , Slasor, Peter and Cohen Pfeffer, Jessica
Publisher
England: Elsevier Limited
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Language
English
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Publisher
England: Elsevier Limited
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Scope and Contents
Contents
Cerliponase alfa is a recombinant human tripeptidyl peptidase 1 (TPP1) enzyme replacement therapy for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2 disease), which is caused by mutations in the TPP1 gene. We aimed to determine the long-term safety and efficacy of intracerebroventricular cerliponase alfa in children with CLN2 disease....
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Full title
Safety and efficacy of cerliponase alfa in children with neuronal ceroid lipofuscinosis type 2 (CLN2 disease): an open-label extension study
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Record Identifier
TN_cdi_proquest_miscellaneous_2902948561
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_2902948561
Other Identifiers
ISSN
1474-4422,1474-4465
E-ISSN
1474-4465
DOI
10.1016/S1474-4422(23)00384-8
