Cerliponase Alfa: First Global Approval

@article{Markham2017CerliponaseAF,
  title={Cerliponase Alfa: First Global Approval},
  author={Anthony Markham},
  journal={Drugs},
  year={2017},
  volume={77},
  pages={1247-1249}
}
  • A. Markham
  • Published 6 June 2017
  • Medicine, Biology
  • Drugs
Cerliponase alfa (Brineura™) is a recombinant human tripeptidyl peptidase-1 (TPP1) being developed by BioMarin Pharmaceutical Inc. for use in patients with neuronal ceroid lipofuscinosis type 2 (CLN2), a paediatric neurodegenerative disease caused by a deficiency in TPP1. CLN2 is characterised by progressive impairment of motor function, language deficiencies, seizures, ataxia, blindness and early death, and intracerebroventricular infusion of cerliponase alfa has been shown to reduce the… 
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