Cerliponase alfa for the treatment of atypical phenotypes of CLN2 disease: A retrospective case series

Eva Wibbeler,Raymond Wang,Emily de los Reyes,Nicola Specchio,Paul Gissen,Norberto Guelbert,Miriam Nickel,Christoph Schwering,Lenora Lehwald,Marina Trivisano,Laura Lee,Gianni Amato,Jessica Cohen-Pfeffer,Renee Shediac,Fernanda Leal-Pardinas,Angela Schulz

MOLECULAR GENETICS AND METABOLISM（2020）

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摘要

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

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关键词

CLN2 disease,atypical,cerliponase alfa,late infantile neuronal ceroid lipofuscinosis,natural history,neuronal ceroid lipofuscinosis type 2

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