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Page 1
Safety and efficacy of arimoclomol in patients with early amyotrophic lateral sclerosis (ORARIALS-01): a randomised, double-blind, placebo-controlled, multicentre, phase 3 trial.
Benatar M, Hansen T, Rom D, Geist MA, Blaettler T, Camu W, Kuzma-Kozakiewicz M, van den Berg LH, Morales RJ, Chio A, Andersen PM, Pradat PF, Lange D, Van Damme P, Mora G, Grudniak M, Elliott M, Petri S, Olney N, Ladha S, Goyal NA, Meyer T, Hanna MG, Quinn C, Genge A, Zinman L, Jabari D, Shoesmith C, Ludolph AC, Neuwirth C, Nations S, Shefner JM, Turner MR, Wuu J, Bennett R, Dang H, Sundgreen C; ORARIALS-01 trial team. Benatar M, et al. Among authors: morales rj. Lancet Neurol. 2024 Jul;23(7):687-699. doi: 10.1016/S1474-4422(24)00134-0. Epub 2024 May 20. Lancet Neurol. 2024. PMID: 38782015 Clinical Trial.
Efficacy, safety and tolerability of rozanolixizumab in patients with chronic inflammatory demyelinating polyradiculoneuropathy: a randomised, subject-blind, investigator-blind, placebo-controlled, phase 2a trial and open-label extension study.
Querol L, De Sèze J, Dysgaard T, Levine T, Rao TH, Rivner M, Hartung HP, Kiessling P, Shimizu S, Marmol D, Bozorg A, Colson AO, Massow U, Eftimov F; CIDP01 Study Investigators. Querol L, et al. J Neurol Neurosurg Psychiatry. 2024 Aug 16;95(9):845-854. doi: 10.1136/jnnp-2023-333112. J Neurol Neurosurg Psychiatry. 2024. PMID: 38729747 Free PMC article. Clinical Trial.
Transthyretin amyloid polyneuropathy in France: A cross-sectional study with 413 patients and real-world tafamidis meglumine use (2009-2019).
Adams D, Cintas P, Solé G, Tard C, Labeyrie C, Echaniz-Laguna A, Cauquil C, Pereon Y, Magy L, Morales RJ, Antoine JC, Lagrange E, Petiot P, Mallaret M, Francou B, Guiochon-Mantel A, Coste A, Demarcq O, Geffroy C, Famelart V, Rudant J, Bartoli M, Donal E, Lairez O, Eicher JC, Kharoubi M, Oghina S, Trochu JN, Inamo J, Habib G, Roubille F, Hagège A, Morio F, Cariou E, Adda J, Slama MS, Charron P, Algalarrondo V, Damy T, Attarian S. Adams D, et al. Among authors: morales rj. Rev Neurol (Paris). 2024 Sep;180(7):661-672. doi: 10.1016/j.neurol.2024.02.393. Epub 2024 Apr 19. Rev Neurol (Paris). 2024. PMID: 38643028
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.
Post-polio syndrome is not a dysimmune condition.
Laffont I, Duflos C, Hirtz C, Bakhti K, Gelis A, Palayer C, Macioce V, Soler M, Pradalier F, Galtier F, Jentzer A, Lozano C, Vincent T, Morales RJ. Laffont I, et al. Among authors: morales rj. Eur J Phys Rehabil Med. 2024 Apr;60(2):270-279. doi: 10.23736/S1973-9087.23.08158-3. Epub 2024 Jan 22. Eur J Phys Rehabil Med. 2024. PMID: 38252127 Free PMC article.
Clinical and genetic features of patients suffering from CMT4J.
Beloribi-Djefaflia S, Morales RJ, Fatehi F, Isapof A, Servais L, Leonard-Louis S, Michaud M, Verdure P, Gidaro T, Pouget J, Poinsignon V, Bonello-Palot N, Attarian S. Beloribi-Djefaflia S, et al. Among authors: morales rj. J Neurol. 2024 Mar;271(3):1355-1365. doi: 10.1007/s00415-023-12076-4. Epub 2023 Nov 11. J Neurol. 2024. PMID: 37950760 Free article.
Titin copy number variations associated with dominant inherited phenotypes.
Perrin A, Métay C, Savarese M, Ben Yaou R, Demidov G, Nelson I, Solé G, Péréon Y, Bertini ES, Fattori F, D'Amico A, Ricci F, Ginsberg M, Seferian A, Boespflug-Tanguy O, Servais L, Chapon F, Lagrange E, Gaudon K, Bloch A, Ghanem R, Guyant-Maréchal L, Johari M, Van Goethem C, Fardeau M, Morales RJ, Genetti CA, Marttila M, Koenig M, Beggs AH, Udd B, Bonne G, Cossée M. Perrin A, et al. Among authors: morales rj. J Med Genet. 2024 Mar 21;61(4):369-377. doi: 10.1136/jmg-2023-109473. J Med Genet. 2024. PMID: 37935568 Free article.
Role of autoantibody levels as biomarkers in the management of patients with myasthenia gravis: A systematic review and expert appraisal.
Meisel A, Baggi F, Behin A, Evoli A, Kostera-Pruszczyk A, Mantegazza R, Morales RJ, Punga AR, Sacconi S, Schroeter M, Verschuuren J, Crathorne L, Holmes K, Leite MI. Meisel A, et al. Among authors: morales rj. Eur J Neurol. 2023 Jan;30(1):266-282. doi: 10.1111/ene.15565. Epub 2022 Oct 7. Eur J Neurol. 2023. PMID: 36094738 Review.
Objective Evaluation of Clinical Actionability for Genes Involved in Myopathies: 63 Genes with a Medical Value for Patient Care.
Vecten M, Pion E, Bartoli M, Morales RJ, Sternberg D, Rendu J, Stojkovic T, Bourdain CA, Métay C, Richard I, Cerino M, Milh M, Campana-Salort E, Gorokhova S, Levy N, Latypova X, Bonne G, Biancalana V, Petit F, Molon A, Perrin A, Laforêt P, Attarian S, Krahn M, Cossée M. Vecten M, et al. Among authors: morales rj. Int J Mol Sci. 2022 Jul 31;23(15):8506. doi: 10.3390/ijms23158506. Int J Mol Sci. 2022. PMID: 35955641 Free PMC article.
35 results