Treatment-related benefit and satisfaction in patients with Fabry disease in France: insight into patients’ expectations and preferences from the prospective, non-interventional SATIS-Fab study

Abstract Background Fabry disease (FD) is a progressive X-linked lysosomal disorder caused by GLA variants resulting in deficient α-galactosidase A enzyme activity, glycolipid accumulation, and multisystemic dysfunction. Approved treatments include intravenous enzyme replacement therapy (ERT) or the...

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Hlavní autoři:	Olivier Lidove, Agathe Masseau, Grégory Pugnet, Didier Lacombe, Bertrand Dussol, Soumeya Bekri, Albert Hagège, Caroline Martinez, Yann Fardini, Alain Fouilhoux, Esther Noël
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	BMC 2026-04-01
Edice:	Orphanet Journal of Rare Diseases
Témata:	Fabry disease Migalastat Enzyme replacement therapy Treatment expectations Treatment satisfaction Patient Benefit Index
On-line přístup:	https://doi.org/10.1186/s13023-026-04285-7
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