Multicentric Retrospective Evaluation of Five Classic Infantile Pompe Disease Subjects Under Enzyme Replacement Therapy With Early Infratentorial Involvement

Multicentric Retrospective Evaluation of Five Classic Infantile Pompe Disease Subjects Under Enzyme Replacement Therapy With Early Infratentorial Involvement

White matter (WM) abnormalities and ventricular enlargement in brain MRI are well-known features in infantile-onset Pompe disease (IOPD) in the era of enzyme replacement therapy (ERT). In this multicentric observational retrospective study, we report a small cohort of IOPD subjects under ERT treatme...

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Bibliographic Details
Main Authors: Matteo Paoletti, Anna Pichiecchio, Giovanna Stefania Colafati, Giorgio Conte, Federica Deodato, Serena Gasperini, Francesca Menni, Francesca Furlan, Laura Rubert, Fabio Maria Triulzi, Claudia Cinnante
Format: Article
Language:English
Published: Frontiers Media S.A. 2020-11-01
Series:Frontiers in Neurology
Subjects:
Pompe disease
IOPD
infantile-onset Pompe disease
brain MRI
ERT (enzyme replacement therapy)
white matter (WM)
Online Access:https://www.frontiersin.org/articles/10.3389/fneur.2020.569153/full

Internet

https://www.frontiersin.org/articles/10.3389/fneur.2020.569153/full

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