QR Kodea

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...

Deskribapen osoa

Gorde:
Egile Nagusiak: Olivier Lidove, Agathe Masseau, Grégory Pugnet, Didier Lacombe, Bertrand Dussol, Soumeya Bekri, Albert Hagège, Caroline Martinez, Yann Fardini, Alain Fouilhoux, Esther Noël
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: BMC 2026-04-01
Saila:Orphanet Journal of Rare Diseases
Gaiak:
Fabry disease
Migalastat
Enzyme replacement therapy
Treatment expectations
Treatment satisfaction
Patient Benefit Index
Sarrera elektronikoa:https://doi.org/10.1186/s13023-026-04285-7
Etiketak: Etiketa erantsi
Etiketarik gabe, Izan zaitez lehena erregistro honi etiketa jartzen!

Antzeko izenburuak