Results 101 to 110 of about 69,398 (132)

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, Emily De Los Reyes
exaly   +2 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.
Anu Cherukuri
exaly   +3 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 ...
CDA-AMC
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   +3 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   +3 more sources

Seizures and Movement Disorders in Patients with CLN2 Disease Treated with Cerliponase Alfa in the Real-World Setting

Neuropediatrics
Angela Schulz, Miriam Nickel, Christoph Schwering, Eva Wibbeler, Lena Marie Westermann, Luca Hagenah, Sheila Reddy, Abigail Hunt, Matthias Hunger, Olivia Okoli, Pascal Reisewitz   +10 more
semanticscholar   +3 more sources

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

Journal of Neurosurgery: Pediatrics, 2022
C. Craven
openaire   +3 more sources

Cerliponase-alfa/Dexamethasone/gentamicin

Reactions Weekly
openaire   +2 more sources

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   +2 more sources

New product intros (brigatinib, cerliponase alfa, niraparib hydrochloride, midostaurin, ocrelizumab, valbenazine)

Drugs of Today, 2017
openaire   +2 more sources