The GALA project: practical recommendations for the use of migalastat in clinical practice on the basis of a structured survey among Italian experts
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,...
Source: Orphanet Journal of Rare Diseases - Category: Internal Medicine Authors: Cristina Chimenti, Patrizia Nencini, Federico Pieruzzi, Sandro Feriozzi, Renzo Mignani, Maurizio Pieroni and Antonio Pisani Tags: Position statement Source Type: research