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Year Number of Results
2020 1
2021 7
2022 9
2023 6
2024 4

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22 results

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Page 1
Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial.
Diaz-Manera J, Kishnani PS, Kushlaf H, Ladha S, Mozaffar T, Straub V, Toscano A, van der Ploeg AT, Berger KI, Clemens PR, Chien YH, Day JW, Illarioshkin S, Roberts M, Attarian S, Borges JL, Bouhour F, Choi YC, Erdem-Ozdamar S, Goker-Alpan O, Kostera-Pruszczyk A, Haack KA, Hug C, Huynh-Ba O, Johnson J, Thibault N, Zhou T, Dimachkie MM, Schoser B; COMET Investigator Group. Diaz-Manera J, et al. Lancet Neurol. 2021 Dec;20(12):1012-1026. doi: 10.1016/S1474-4422(21)00241-6. Lancet Neurol. 2021. PMID: 34800399 Clinical Trial.
Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial.
Kishnani PS, Diaz-Manera J, Toscano A, Clemens PR, Ladha S, Berger KI, Kushlaf H, Straub V, Carvalho G, Mozaffar T, Roberts M, Attarian S, Chien YH, Choi YC, Day JW, Erdem-Ozdamar S, Illarioshkin S, Goker-Alpan O, Kostera-Pruszczyk A, van der Ploeg AT, An Haack K, Huynh-Ba O, Tammireddy S, Thibault N, Zhou T, Dimachkie MM, Schoser B; COMET Investigator Group. Kishnani PS, et al. JAMA Neurol. 2023 Jun 1;80(6):558-567. doi: 10.1001/jamaneurol.2023.0552. JAMA Neurol. 2023. PMID: 37036722 Free PMC article. Clinical Trial.
Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial.
Schoser B, Roberts M, Byrne BJ, Sitaraman S, Jiang H, Laforêt P, Toscano A, Castelli J, Díaz-Manera J, Goldman M, van der Ploeg AT, Bratkovic D, Kuchipudi S, Mozaffar T, Kishnani PS; PROPEL Study Group. Schoser B, et al. Lancet Neurol. 2021 Dec;20(12):1027-1037. doi: 10.1016/S1474-4422(21)00331-8. Lancet Neurol. 2021. PMID: 34800400 Clinical Trial.
Drug-refractory myasthenia gravis: Clinical characteristics, treatments, and outcome.
Cortés-Vicente E, Álvarez-Velasco R, Pla-Junca F, Rojas-Garcia R, Paradas C, Sevilla T, Casasnovas C, Gómez-Caravaca MT, Pardo J, Ramos-Fransi A, Pelayo-Negro AL, Gutiérrez-Gutiérrez G, Turon-Sans J, López de Munain A, Guerrero-Sola A, Jericó I, Martín MA, Mendoza MD, Morís G, Vélez-Gómez B, Garcia-Sobrino T, Pascual-Goñi E, Reyes-Leiva D, Illa I, Gallardo E. Cortés-Vicente E, et al. Among authors: reyes leiva d. Ann Clin Transl Neurol. 2022 Feb;9(2):122-131. doi: 10.1002/acn3.51492. Epub 2022 Jan 26. Ann Clin Transl Neurol. 2022. PMID: 35080153 Free PMC article.
Neuroinflammation-Related Proteins NOD2 and Spp1 Are Abnormally Upregulated in Amyotrophic Lateral Sclerosis.
de Luna N, Carbayo Á, Dols-Icardo O, Turon-Sans J, Reyes-Leiva D, Illan-Gala I, Jericó I, Pagola-Lorz I, Lleixà C, Querol L, Rubio-Guerra S, Alcolea D, Fortea J, Lleó A, Cortés-Vicente E, Rojas-Garcia R. de Luna N, et al. Among authors: reyes leiva d. Neurol Neuroimmunol Neuroinflamm. 2022 Dec 2;10(2):e200072. doi: 10.1212/NXI.0000000000200072. Print 2023 Mar. Neurol Neuroimmunol Neuroinflamm. 2022. PMID: 36460480 Free PMC article.
Pathophysiological Underpinnings of Extra-Motor Neurodegeneration in Amyotrophic Lateral Sclerosis: New Insights From Biomarker Studies.
Reyes-Leiva D, Dols-Icardo O, Sirisi S, Cortés-Vicente E, Turon-Sans J, de Luna N, Blesa R, Belbin O, Montal V, Alcolea D, Fortea J, Lleó A, Rojas-García R, Illán-Gala I. Reyes-Leiva D, et al. Front Neurol. 2022 Jan 18;12:750543. doi: 10.3389/fneur.2021.750543. eCollection 2021. Front Neurol. 2022. PMID: 35115992 Free PMC article. Review.
Reduced Number of Thymoma CTLA4-Positive Cells Is Associated With a Higher Probability of Developing Myasthenia Gravis.
Álvarez-Velasco R, Dols-Icardo O, El Bounasri S, López-Vilaró L, Trujillo JC, Reyes-Leiva D, Suárez-Calvet X, Cortés-Vicente E, Illa I, Gallardo E. Álvarez-Velasco R, et al. Among authors: reyes leiva d. Neurol Neuroimmunol Neuroinflamm. 2023 Jan 25;10(2):e200085. doi: 10.1212/NXI.0000000000200085. Print 2023 Mar. Neurol Neuroimmunol Neuroinflamm. 2023. PMID: 36697230 Free PMC article.
Description of clinical and genetic features of 122 patients included in the Spanish Pompe registry.
Martinez-Marin RJ, Reyes-Leiva D, Nascimento A, Muelas N, Dominguez-González C, Paradas C, Olivé M, García-Romero M, Pascual-Pascual SI, Grau JM, Barba-Romero MA, Gomez-Caravaca MT, de Las Heras J, Casquero P, Mendoza MD, de León JC, Gutierrez A, Morís G, Blanco-Lago R, Ramos-Fransi A, Pintós G, García-Antelo MJ, Rabasa M, Morgado Y, Usón M, Miralles FJ, Bárcena-Llona JE, Gómez-Belda AB, Pedraza-Hueso MI, Hortelano M, Colomé A, Garcia-Martin G, Lopez de Munain A, Jericó I, Galán-Dávila L, Pardo J, Salgueiro-Origlia G, Alonso-Pérez J, Pla-Junca F, Schiava M, Segovia-Simón S, Díaz-Manera J. Martinez-Marin RJ, et al. Among authors: reyes leiva d. Neuromuscul Disord. 2024 Jan;34:1-8. doi: 10.1016/j.nmd.2023.10.001. Epub 2023 Nov 13. Neuromuscul Disord. 2024. PMID: 38087756
104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07).
Schoser B, Kishnani PS, Bratkovic D, Byrne BJ, Claeys KG, Díaz-Manera J, Laforêt P, Roberts M, Toscano A, van der Ploeg AT, Castelli J, Goldman M, Holdbrook F, Sitaraman Das S, Wasfi Y, Mozaffar T; ATB200-07 Study Group. Schoser B, et al. J Neurol. 2024 May;271(5):2810-2823. doi: 10.1007/s00415-024-12236-0. Epub 2024 Feb 28. J Neurol. 2024. PMID: 38418563 Free PMC article. Clinical Trial.
22 results