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Page 1
Renal and multisystem effectiveness of 3.9 years of migalastat in a global real-world cohort: Results from the followME Fabry Pathfinders registry.
Hughes DA, Sunder-Plassmann G, Jovanovic A, Brand E, West ML, Bichet DG, Pisani A, Nowak A, Torra R, Khan A, Azevedo O, Lehman A, Linhart A, Rutecki J, Giuliano JD, Krusinska E, Nordbeck P. Hughes DA, et al. J Inherit Metab Dis. 2025 Jan;48(1):e12771. doi: 10.1002/jimd.12771. Epub 2024 Jul 19. J Inherit Metab Dis. 2025. PMID: 39031114 Free PMC article.
Real-life effectiveness 1 year after switching to avalglucosidase alfa in late-onset Pompe disease patients worsening on alglucosidase alfa therapy: A French cohort study.
Tard C, Bouhour F, Michaud M, Beltran S, Fournier M, Demurger F, Lagrange E, Nollet S, Sacconi S, Noury JB, Magot A, Cintas P, Renard D, Deibener-Kaminsky J, Lefeuvre C, Davion JB, Salort-Campana E, Arrassi A, Taouagh N, Spinazzi M; Pompe Study Group; Attarian S, Laforêt P. Tard C, et al. Eur J Neurol. 2024 Jul;31(7):e16292. doi: 10.1111/ene.16292. Epub 2024 Apr 8. Eur J Neurol. 2024. PMID: 38587143 Free PMC article.
Inflammation and Exosomes in Fabry Disease Pathogenesis.
Coelho-Ribeiro B, Silva HG, Sampaio-Marques B, Fraga AG, Azevedo O, Pedrosa J, Ludovico P. Coelho-Ribeiro B, et al. Cells. 2024 Apr 9;13(8):654. doi: 10.3390/cells13080654. Cells. 2024. PMID: 38667269 Free PMC article. Review.
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, Sitaraman Das S, Goldman M, Mozaffar T; PROPEL Study Group. Kushlaf H, et al. Muscle Nerve. 2025 May 7. doi: 10.1002/mus.28420. Online ahead of print. Muscle Nerve. 2025. PMID: 40342075
Context-Specific Function of the Engineered Peptide Domain of PHP.B.
Martino RA, Fluck EC 3rd, Murphy J, Wang Q, Hoff H, Pumroy RA, Lee CY, Sims JJ, Roy S, Moiseenkova-Bell VY, Wilson JM. Martino RA, et al. J Virol. 2021 Sep 27;95(20):e0116421. doi: 10.1128/JVI.01164-21. Epub 2021 Aug 4. J Virol. 2021. PMID: 34346767 Free PMC article.
16 results