Last Updated: 09/26/2026
Save publications for later
Sign Up
Not sure about your diagnosis?
Check Your Symptoms
Found 6 publications
Clinical Pharmacokinetics and Pharmacodynamics of Cerliponase Alfa, Enzyme Replacement Therapy for CLN2 Disease by Intracerebroventricular Administration.
Journal: Clinical and translational science
Published: August 04, 2020
Neurofilament light is a treatment-responsive biomarker in CLN2 disease.
Journal: Annals of clinical and translational neurology
Published: October 03, 2019
Regulatory strategies for rare diseases under current global regulatory statutes: a discussion with stakeholders.
Journal: Orphanet journal of rare diseases
Published: November 28, 2018
Study of Intraventricular Cerliponase Alfa for CLN2 Disease.
Journal: The New England journal of medicine
Published: April 25, 2018
Disease characteristics and progression in patients with late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease: an observational cohort study.
Journal: The Lancet. Child & adolescent health
Published: March 30, 2018
Showing 1-6 of 6
Last Updated: 09/26/2026