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The emerging phenotype of long-term survivors with infantile Pompe disease
PURPOSE: Enzyme replacement therapy with alglucosidase alfa for infantile Pompe disease has improved survival creating new management challenges. We describe an emerging phenotype in a retrospective review of long-term survivors. METHODS: Inclusion criteria included ventilator-free status and age ≤6...
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| Main Authors: | , , , , , , , , , , |
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| Format: | Artigo |
| Jezik: | Inglês |
| Izdano: |
2012
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| Teme: | |
| Online dostop: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3947503/ https://ncbi.nlm.nih.gov/pubmed/22538254 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gim.2012.44 |
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