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Fabry Disease: Multidisciplinary Evaluation After 10 Years of Treatment with Agalsidase Beta
Fabry disease is an X linked disorder of metabolism due to deficient α-galactosidase A activity. Enzyme replacement therapy (ERT) with agalsidase Beta was approved by EMA in 2001 and FDA in 2003. Patients and methods: Six patients were enrolled. Baseline data was measured for renal, cardiac, and cer...
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| I publikationen: | JIMD Rep |
|---|---|
| Huvudupphovsmän: | , , , , , , , , , , |
| Materialtyp: | Artigo |
| Språk: | Inglês |
| Publicerad: |
Springer Berlin Heidelberg
2014
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| Ämnen: | |
| Länkar: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4221298/ https://ncbi.nlm.nih.gov/pubmed/24850233 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/8904_2014_310 |
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