A favorable outcome in an infantile-onset Pompe patient with cross reactive immunological material (CRIM) negative disease with high dose enzyme replacement therapy and adjusted immunomodulation

Infantile onset Pompe disease (IOPD) is a rare devastating disease that presents in early infancy with rapidly progressive hypertrophic cardiomyopathy, severe generalized myopathy and death within the first year of life. The emergence of enzyme replacement therapy (ERT) with recombinant human acid a...

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Detalles Bibliográficos
Autores principales: Curelaru, Shiri, Desai, Ankit K., Fink, Daniel, Zehavi, Yoav, Kishnani, Priya S., Spiegel, Ronen
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2022
Materias:
Research Paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9263520/
https://www.ncbi.nlm.nih.gov/pubmed/35813979
http://dx.doi.org/10.1016/j.ymgmr.2022.100893

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9263520/
https://www.ncbi.nlm.nih.gov/pubmed/35813979
http://dx.doi.org/10.1016/j.ymgmr.2022.100893

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