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Economic analysis of cerliponase alfa for treatment of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2)

Expert Review of Pharmacoeconomics and Outcomes Research, 2023
Cerliponase alfa is an orphan drug approved for the treatment of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2).Our goal was to assess the cost-effectiveness of cerliponase alfa in patients with CLN2 in the socioeconomic context of the Republic of Serbia in contrast to symptomatic therapy.For this study, a forty-year horizon and the ...
Slobodan Janković, Miloš Milosavljevic, Teodora Safiye   +2 more
exaly   +4 more sources

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Cerliponase alfa for CLN2 disease, a promising therapy

Expert Opinion on Orphan Drugs, 2020
Introduction: Neuronal ceroid lipofuscinosis type 2 (CLN2) is a rare, lysosomal storage disease that causes progressive neurodegeneration in children.
Nicolas J Abreu, Jonathan Pindrik
exaly   +2 more sources

Immunogenicity to cerliponase alfa intracerebroventricular enzyme replacement therapy for CLN2 disease: Results from a Phase 1/2 study

Clinical Immunology, 2018
Treatment with intracerebroventricular (ICV)-delivered cerliponase alfa enzyme replacement therapy (ERT) in a Phase 1/2 study of 24 subjects with CLN2 disease resulted in a meaningful preservation of motor and language (ML) function and was well tolerated. Treatment was associated with anti-drug antibody (ADA) production in the cerebrospinal fluid (CSF)
Andrea Van Tuyl, Becky Schweighardt, Peter Slasor   +2 more
exaly   +3 more sources

Cerliponase alfa decreases Aβ load and alters autophagy- related pathways in mouse hippocampal neurons exposed to fAβ1–42

Life Sciences
Extracellular aggregation of amyloid-beta (Aβ) in the brain plays a central role in the onset and progression of Alzheimer's disease (AD). Moreover, intraneuronal accumulation of Aβ via oligomer internalization might play an important role in the progression of AD.
Pelin Kelicen Uğur, Hulya Karatas, Hilal Akyel   +2 more
exaly   +5 more sources

Safety and efficacy of cerliponase alfa in children with neuronal ceroid lipofuscinosis type 2 (CLN2 disease): an open-label extension study [PDF]

Lancet Neurology, The
Background: 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.
Peter Slasor
exaly   +2 more sources

Cerliponase-alfa

Reactions Weekly, 2021
openaire   +2 more sources

Review of Cerliponase Alfa: Recombinant Human Enzyme Replacement Therapy for Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2

Journal of Child Neurology, 2019
The objective of this review is to summarize the pharmacology, efficacy, and safety of cerliponase alfa for the treatment of late infantile neuronal ceroid lipofuscinosis type 2 (CLN2). Cerliponase alfa is recombinant human tripeptidyl peptidase 1 enzyme replacement therapy.
Grace Lewis, Amanda M. Morrill, Stephanie L. Conway-Allen, Bernard Kim   +3 more
openaire   +2 more sources