Results 71 to 80 of about 392 (96)

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

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   +3 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