Two-year follow-up of gait and postural control following initiation of recombinant human tripeptidy...
Two-year follow-up of gait and postural control following initiation of recombinant human tripeptidyl intracerebroventricular enzyme replacement therapy in two atypical CLN2 patients
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London: Nature Publishing Group UK
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English
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London: Nature Publishing Group UK
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Neuronal ceroid lipofuscinosis type 2 (CLN2) is a rapidly progressive neurodegenerative disorder leading to premature mortality. Ambulatory CLN2 patients typically receive standard of care treatment through biweekly intracerebroventricular (ICV) enzyme replacement therapy (ERT) involving recombinant human tripeptidyl peptidase 1, known as cerlipona...
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Full title
Two-year follow-up of gait and postural control following initiation of recombinant human tripeptidyl intracerebroventricular enzyme replacement therapy in two atypical CLN2 patients
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TN_cdi_doaj_primary_oai_doaj_org_article_6f7efd353b754881babb6a87f0382c2a
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_6f7efd353b754881babb6a87f0382c2a
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ISSN
2045-2322
E-ISSN
2045-2322
DOI
10.1038/s41598-024-82157-5
