Characterization of immune response in Cross-Reactive Immunological Material (CRIM)-positive infantile Pompe disease patients treated with enzyme replacement therapy

Enzyme replacement therapy (ERT) with rhGAA has improved clinical outcomes in infantile Pompe disease (IPD). A subset of CRIM-positive IPD patients develop high and sustained antibody titers (HSAT; ≥51,200) and/or sustained intermediate titer (SIT; ≥12,800 and <51,200), similar to CRIM-negative p...

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Detalles Bibliográficos
Autores principales: Desai, Ankit K., Kazi, Zoheb B., Bali, Deeksha S., Kishnani, Priya S.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2019
Materias:
Research Paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6518314/
https://www.ncbi.nlm.nih.gov/pubmed/31193175
http://dx.doi.org/10.1016/j.ymgmr.2019.100475

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