Study of intraventricular cerliponase alfa for CLN2 disease Academic Article uri icon

Overview

MeSH Major

  • Dipeptidyl-Peptidases and Tripeptidyl-Peptidases
  • Enzyme Replacement Therapy
  • Neuronal Ceroid-Lipofuscinoses
  • Recombinant Proteins

abstract

  • Intraventricular infusion of cerliponase alfa in patients with CLN2 disease resulted in less decline in motor and language function than that in historical controls. Serious adverse events included failure of the intraventricular device and device-related infections. (Funded by BioMarin Pharmaceutical and others; CLN2 ClinicalTrials.gov numbers, NCT01907087 and NCT02485899 .).

publication date

  • May 17, 2018

Research

keywords

  • Academic Article

Identity

Language

  • eng

Digital Object Identifier (DOI)

  • 10.1056/NEJMoa1712649

PubMed ID

  • 29688815

Additional Document Info

start page

  • 1898

end page

  • 1907

volume

  • 378

number

  • 20