Neurofilament Light and Its Association With CNS Involvement in Patients With Classic Infantile Pompe Disease

BACKGROUND AND OBJECTIVES: Enzyme replacement therapy (ERT) has substantially improved the outcome of classic infantile Pompe disease, an inheritable muscle disease previously fatal at infancy. However, under treatment, patients develop white matter abnormalities and neurocognitive problems. Therefo...

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Detalles Bibliográficos
Autores principales: Mackenbach, Maarten J., Willemse, Eline A.J., van den Dorpel, Jan J.A., van der Beek, Nadine A.M.E., Díaz-Manera, Jordi, Rizopoulos, Dimitris, Teunissen, Charlotte, van der Ploeg, Ans T., van den Hout, Johanna M.P.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Lippincott Williams & Wilkins 2023
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10424841/
https://www.ncbi.nlm.nih.gov/pubmed/37336766
http://dx.doi.org/10.1212/WNL.0000000000207482

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10424841/
https://www.ncbi.nlm.nih.gov/pubmed/37336766
http://dx.doi.org/10.1212/WNL.0000000000207482

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