Cargando…
Immune responses to alglucosidase in infantile Pompe disease: recommendations from an Italian pediatric expert panel
BACKGROUND: Classic infantile onset of Pompe disease (c-IOPD) leads to hypotonia and hypertrophic cardiomyopathy within the first days to weeks of life and, without treatment, patients die of cardiorespiratory failure in their first 1–2 years of life. Enzymatic replacement therapy (ERT) with algluco...
Autores principales: | , , , , , , , , , , , , , |
---|---|
Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2022
|
Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8898438/ https://www.ncbi.nlm.nih.gov/pubmed/35248118 http://dx.doi.org/10.1186/s13052-022-01219-4 |