Economic analysis of cerliponase alfa for treatment of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2).

Medo GutićAleksandra Dimitrijević Teodora SafiyeArdea MilidragSlobodan M Jankovic

Published in: Expert review of pharmacoeconomics & outcomes research (2023)

Cerliponasealfa is not more economical than symptomatic therapy for the treatment of CLN2when using typical pharmacoeconomic analysis. Cerliponase alfa has been shownto be effective, but more has to be done to make it accessible to all CLN2patients.

Keyphrases

end stage renal disease
replacement therapy
newly diagnosed
ejection fraction
peritoneal dialysis
prognostic factors
combination therapy
blood brain barrier