Adverse Reactions to the Orphan Drug Cerliponase Alfa in the Treatment of Neurolipofuscinosis Type 2 (CLN2)

Adverse Reactions to the Orphan Drug Cerliponase Alfa in the Treatment of Neurolipofuscinosis Type 2 (CLN2)

<b>Background/Objectives:</b> Neuronal Ceroid Lipofuscinosis type 2 is a rare pathology affecting mainly the central nervous system (CNS) and retina, and is caused by variants in the gene encoding the lysosomal enzyme tripeptidyl peptidase 1. Therapy with enzyme replacement through the b...

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Main Authors: Ilaria Ammendolia (Author), Maria Sframeli (Author), Emanuela Esposito (Author), Luigi Cardia (Author), Alberto Noto (Author), Mariaconcetta Currò (Author), Gioacchino Calapai (Author), Maria De Pasquale (Author), Carmen Mannucci (Author), Fabrizio Calapai (Author)
Format: Book
Published: MDPI AG, 2024-11-01T00:00:00Z.
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3rd Floor Main Library

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