The GALA project: practical recommendations for the use of migalastat in clinical practice on the basis of a structured survey among Italian experts

The GALA project: practical recommendations for the use of migalastat in clinical practice on the basis of a structured survey among Italian experts

Abstract 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 impo...

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Bibliographic Details
Main Authors: Cristina Chimenti, Patrizia Nencini, Federico Pieruzzi, Sandro Feriozzi, Renzo Mignani, Maurizio Pieroni, Antonio Pisani, on behalf of the GALA Working Group
Format: Article
Language:English
Published: BMC 2020-04-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Fabry disease
Migalastat
Treatment
Enzyme-replacement therapy
Expert opinion
Online Access:http://link.springer.com/article/10.1186/s13023-020-1318-8

Internet

http://link.springer.com/article/10.1186/s13023-020-1318-8

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