Page 194 - MEMORIA_SEHH-FEHH_2023

MEMORIA ANUAL 2023 GEEDL
SOCIEDAD ESPAÑOLA DE
HEMATOLOGÍA Y HEMOTERAPIA
FUNDACIÓN ESPAÑOLA DE
HEMATOLOGÍA Y HEMOTERAPIA
Publicaciones en revistas científicas
Internacionales
• Advantages of digital technology in the assessment of bone marrow involvement in
Gaucher’s disease.
Front Med (Lausanne). 2023;10:1098472. doi: 10.3389/fmed.2023.1098472.
Valero-Tena E, Roca-Espiau M, Verdú-Díaz J, Diaz-Manera J, Andrade-Campos M, Gi-
raldo P.
• Management goals of type 1 Gaucher disease in South Africa: An expert Delphi con-
sensus document on good clinical practice.
PLoS One. 2023;18(8):e0290401. doi: 10.1371/journal.pone.0290401.
Louw VJ, Fraser I, Giraldo P.
• Long-term safety and efficacy of pegunigalsidase alfa: A multicenter 6-year study in
adult patients with Fabry disease.
Genet Med. 2023;25(12):100968. doi: 10.1016/j.gim.2023.100968.
Hughes D, González D, Maegawa G, Bernat JA, Holida M, Giraldo P, Atta MG, Chertkoff R,
Alon S, Almon EB, Rocco R, Goker-Alpan O.
• Safety and efficacy of pegunigalsidase alfa in patients with Fabry disease who were
previously treated with agalsidase alfa: results from BRIDGE, a phase 3 open-label
study.
Orphanet J Rare Dis. 2023;18(1):332. doi: 10.1186/s13023-023-02937-6.
Linhart A, Dostálová G, Nicholls K, West ML, Tøndel C, Jovanovic A, Giraldo P, Vujkovac B,
Geberhiwot T, Brill-Almon E, Alon S, Chertkoff R, Rocco R, Hughes D.
• Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with
Fabry disease and deteriorating renal function: results from the 2-year randomised
phase III BALANCE study.
J Med Genet. 2023:jmg-2023-109445. doi: 10.1136/jmg-2023-109445.
Wallace EL, Goker-Alpan O, Wilcox WR, Holida M, Bernat J, Longo N, Linhart A, Hughes
DA, Hopkin RJ, Tøndel C, Langeveld M, Giraldo P, Pisani A, Germain DP, Mehta A, Dee-
gan PB, Molnar MJ, Ortiz D, Jovanovic A, Muriello M, Barshop BA, Kimonis V, Vujkovac B,
Nowak A, Geberhiwot T, Kantola I, Knoll J, Waldek S, Nedd K, Karaa A, Brill-Almon E, Alon
S, Chertkoff R, Rocco R, Sakov A, Warnock DG.
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