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01/09/2025
Real-World Multinational Survey of Chronic Inflammatory Demyelinating Polyneuropathy: Disease Characteristics and Therapeutic Landscape
Querol, L, Rinaldi, S, Borsi, A, Boggia, GM, de Courcy, J, Taylor, Y, Wright, J, Karmous, W, Noel, W, Gary, C, zu Hörste, GM. Real-World Multinational Survey of Chronic Inflammatory Demyelinating Polyneuropathy: Disease Characteristics and Therapeutic Landscape. JOURNAL OF THE PERIPHERAL NERVOUS SYSTEM. 2025; 30(3)DOI:10.1111/jns.70047. PMID:40826893. IF:3.200(Q2/4D).
01/09/2025
Hereditary transthyretin amyloidosis caused by the Val142Ile variant in Spain
de Frutos, F, Herrador, L, Peiró-Aventin, B, Eiros, R, Freire, JL, Zorio, E, Carbayo, A, Llongueras-Espi, P, Garcia-Alvarez, A, Ripoll-Vera, T, Macias, R, Vilches, S, Ruiz-Bustillo, S, Arana-Achaga, X, Ordás, JG, Piqueras-Flores, J, Ruiz-Cueto, M, Casasnovas, C, Tirón, C, Rojas-Garcia, R, Sevilla, T, Rodriguez-Palomares, JF, González-López, E, Villacorta, E, Garcia-Pavia, P, González-Costello, J. Hereditary transthyretin amyloidosis caused by the Val142Ile variant in Spain. REVISTA ESPANOLA DE CARDIOLOGIA. 2025; 78(9)DOI:10.1016/j.rec.2024.12.012. PMID:39827963. IF:4.900(Q1/2D).
12/08/2025
Efficacy of Switching Therapy From Alglucosidase Alfa to Avalglucosidase Alfa on Respiratory Function in Participants With Late-Onset Pompe Disease: A Post Hoc Analysis From the COMET Trial
Kishnani, PS, Boentert, M, Wenninger, S, Berger, KI, Msihid, J, O'Callaghan, L, Essadi-Addou, R, Gallego, V, Rawat, NS, Huynh-Ba, O, Diaz-Manera, J. Efficacy of Switching Therapy From Alglucosidase Alfa to Avalglucosidase Alfa on Respiratory Function in Participants With Late-Onset Pompe Disease: A Post Hoc Analysis From the COMET Trial. Jimd Reports. 2025; 66(5)DOI:10.1002/jmd2.70033. PMID:40799512. IF:(Q/D).
01/08/2025
Switching Enzyme Replacement Therapy for Late-Onset Pompe Disease From Alglucosidase Alfa to Cipaglucosidase Alfa Plus Miglustat: Post Hoc Effect Size Analysis of PROPEL
Kushlaf, H, Díaz-Manera, J, Bratkovic, D, Byrne, BJ, Claeys, KG, Clemens, PR, Dimachkie, MM, Kishnani, PS, Laforêt, P, Roberts, M, Schoser, B, Toscano, A, Castelli, J, Holdbrook, F, Das, SS, Goldman, M, Mozaffar, T. Switching Enzyme Replacement Therapy for Late-Onset Pompe Disease From Alglucosidase Alfa to Cipaglucosidase Alfa Plus Miglustat: Post Hoc Effect Size Analysis of PROPEL. MUSCLE & NERVE. 2025; 72(2)DOI:10.1002/mus.28420. PMID:40342075. IF:3.100(Q2/4D).