Open AccessStudy of Intraventricular Cerliponase Alfa for CLN2 Disease
Author(s) -
Angela Schulz,
Temitayo Ajayi,
Nicola Specchio,
Emily de los Reyes,
Paul Gissen,
Douglas Ballon,
Jonathan P. Dyke,
Heather Cahan,
Peter Slasor,
David Jacoby,
Alfried Kohlschütter
Publication year - 2018
Publication title -
new england journal of medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 19.889
H-Index - 1030
eISSN - 1533-4406
pISSN - 0028-4793
DOI - 10.1056/nejmoa1712649
Subject(s) - enzyme replacement therapy , neuronal ceroid lipofuscinosis , medicine , recombinant dna , disease , batten disease , biology , biochemistry , gene
Recombinant human tripeptidyl peptidase 1 (cerliponase alfa) is an enzyme-replacement therapy that has been developed to treat neuronal ceroid lipofuscinosis type 2 (CLN2) disease, a rare lysosomal disorder that causes progressive dementia in children.
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