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The GALA project: practical recommendations for the use of migalastat in clinical practice on the basis of a structured survey among Italian experts
BACKGROUND: Oral migalastat has recently been approved for the treatment of Anderson-Fabry disease (FD) in patients aged ≥16 years with amenable mutations on the basis of two phase III trials, FACETS and ATTRACT. However, with the introduction of migalastat into clinical practice, it is important to...
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| Publicado no: | Orphanet J Rare Dis |
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| Main Authors: | , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
BioMed Central
2020
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7140546/ https://ncbi.nlm.nih.gov/pubmed/32264911 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-020-1318-8 |
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