Results 101 to 110 of about 165,283 (121)

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

Journal of Neurosurgery: Pediatrics, 2022
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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
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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
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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
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Cerliponase alfa not recommended by NICE for Batten disease

PharmacoEconomics & Outcomes News, 2019
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Cerliponase-alfa/Dexamethasone/gentamicin

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

Journal of Child Neurology, 2020
Grace Lewis
exaly  

Cerliponase alfa-related AEs reported in EudraVigilance

Reactions Weekly
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