Cerliponase Alfa for the Treatment of Atypical Phenotypes of CLN2 Disease: A Retrospective Case Series.

Eva WibbelerRaymond WangEmily de Los ReyesNicola SpecchioPaul GissenNorberto GuelbertMiriam NickelChristoph SchweringLenora LehwaldMarina TrivisanoLaura LeeGianni AmatoJessica Cohen-PfefferRenée ShediacFernanda Leal-PardinasAngela Schulz

Published in: Journal of child neurology (2020)

Cerliponase alfa is well tolerated and has the potential to stabilize motor and language function in patients with atypical phenotypes of CLN2 disease.

Keyphrases

replacement therapy
autism spectrum disorder
combination therapy
human health
smoking cessation