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...

وصف كامل

التفاصيل البيبلوغرافية

المؤلفون الرئيسيون:	Olivier Lidove, Agathe Masseau, Grégory Pugnet, Didier Lacombe, Bertrand Dussol, Soumeya Bekri, Albert Hagège, Caroline Martinez, Yann Fardini, Alain Fouilhoux, Esther Noël
التنسيق:	Artigo
اللغة:	Inglês
منشور في:	BMC 2026-04-01
سلاسل:	Orphanet Journal of Rare Diseases
الموضوعات:	Fabry disease Migalastat Enzyme replacement therapy Treatment expectations Treatment satisfaction Patient Benefit Index
الوصول للمادة أونلاين:	https://doi.org/10.1186/s13023-026-04285-7
الوسوم:	إضافة وسم لا توجد وسوم, كن أول من يضع وسما على هذه التسجيلة!