Results 81 to 90 of about 392 (96)

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   +4 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

Cerliponase Alfa for Pediatric Patients With Neuronal Ceroid Lipofuscinosis Type 2 Disease

Canadian Journal of Health Technologies
What Is the Issue? Neuronal ceroid lipofuscinosis type 2 (CLN2) disease is an ultrarare, severe, and rapidly progressing lysosomal storage disorder, with a global incidence of approximately 0.15 to 9.0 per 100,000 live births. It has a devastating impact on children and families, leading to rapid functional decline and early death without effective ...
openaire   +1 more source

Erratum. A survival analysis of ventricular access devices for delivery of cerliponase alfa

Journal of Neurosurgery: Pediatrics, 2022
openaire   +2 more sources

Chest-sited intraventricular access devices for cerliponase alfa infusion in Batten disease at a single tertiary United Kingdom pediatric center

Journal of Neurosurgery: Pediatrics
OBJECTIVE Cerliponase alfa is an enzyme replacement treatment for neuronal ceroid lipofuscinosis type 2 (CLN2), administered via biweekly intracerebroventricular infusions. Typically, infusions are delivered via a head-sited access device; however, subcutaneously tunneled chest-sited devices could offer an ...
Jack, Read, Aimee, Donald, Stephanie, Rhead, Lervia, Acquaah, Gabrielle, Chan, Fiona, Heap, Bethany, Brown, Arunabha, Ghosh, Simon Allan, Jones, Dipak, Ram, Ian, Kamaly-Asl   +10 more
openaire   +2 more sources

Comparing developmental outcomes of children with CLN2 disease receiving cerliponase alfa to a natural history cohort

Molecular Genetics and Metabolism, 2021
Jessica Scherr, Elizabeth Berry-Kravis, Emily de los Reyes   +2 more
openaire   +1 more source

Intracerebroventricular cerliponase alfa for CLN2 disease: Clinical practice considerations from US clinics

Molecular Genetics and Metabolism, 2020
Raymond Y. Wang, Emily de los Reyes, Lenora Lehwald, Erika Augustine, Elizabeth Berry-Kravis, Karen Butler, Natalie Cormier, Scott Demarest, Sam Lu, Jacqueline Madden, Joffre Olaya, Susan See, Amy Vierhile, James Wheless, Amy Yang, Jessica Cohen-Pfeffer, Dorna Chu, Fernanda Leal-Pardinas   +17 more
openaire   +1 more source

Cerliponase Alfa for the Treatment of CLN2 Disease in a Patient Cohort Including Children under 3 Years

Neuropediatrics, 2023
A. Schulz, E. de los Reyes, N. Specchio, P. Gissen, P. Slasor, S. Bondade, J. Cohen-Pfeffer   +6 more
openaire   +1 more source

Cerliponase alfa not recommended by NICE for Batten disease

PharmacoEconomics & Outcomes News, 2019
openaire   +1 more source