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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...
Autores principales: | Chimenti, Cristina, Nencini, Patrizia, Pieruzzi, Federico, Feriozzi, Sandro, Mignani, Renzo, Pieroni, Maurizio, Pisani, Antonio |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7140546/ https://www.ncbi.nlm.nih.gov/pubmed/32264911 http://dx.doi.org/10.1186/s13023-020-1318-8 |
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