Results 121 to 130 of about 69,398 (132)

Serial brain MRI volumetrics and DTI analysis in atypical CLN2 patients treated with ICV cerliponase alfa

Molecular Genetics and Metabolism
Sumiko Abe, Harriet Chang, Audrey Kao, Maija Steenari, Raymond Y. Wang   +4 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

Real-world clinical outcomes of intraventricular cerliponase alfa in CLN2 disease: 4.5-year update from an independent ongoing observational study

Molecular Genetics and Metabolism, 2022
A. Schulz, C. Schwering, E. Wibbeler, L. Westermann, S. Dulz, S. Lezius, M. Nickel   +6 more
semanticscholar   +1 more source

Prospective longitudinal analysis of cardiac function in patients with CLN2 disease under enzyme replacement therapy (ICV-ERT) with cerliponase alfa

Molecular Genetics and Metabolism
A. Schulz, P. Schneider, L. Mamudi, M. Nickel, C. Schwering, F. Stute   +5 more
semanticscholar   +1 more source

Cerliponase alfa not recommended by NICE for Batten disease

PharmacoEconomics & Outcomes News, 2019
openaire   +1 more source

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  

Does Intraventricular Enzyme Replacement Therapy (ICV-ERT) with Cerliponase Alfa Affect Cardiac Function in Patients with CLN2 Disease?

Neuropediatrics
P. Schneider, L. Mamudi, S. Lezius, M. Nickel, C. Schwering, J. Olfe, A. Schulz, F. Stute   +7 more
semanticscholar   +1 more source

Analysis of drug-specific antibody response against cerliponase alfa in CLN2 patients by applying a novel two-step assay

Molecular Genetics and Metabolism
L. Westermann, Zoe-Isabella Junginger, Anna Mann, Eva Tolosa, A. Schulz   +4 more
semanticscholar   +1 more source

Cerliponase Alfa: First Global Approval

Drugs, 2017
Anthony Markham
exaly