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Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry.
Servais L, Day JW, De Vivo DC, Kirschner J, Mercuri E, Muntoni F, Proud CM, Shieh PB, Tizzano EF, Quijano-Roy S, Desguerre I, Saito K, Faulkner E, Benguerba KM, Raju D, LaMarca N, Sun R, Anderson FA, Finkel RS. Servais L, et al. Among authors: saito k. J Neuromuscul Dis. 2024;11(2):425-442. doi: 10.3233/JND-230122. J Neuromuscul Dis. 2024. PMID: 38250783 Free PMC article.
Combination disease-modifying treatment in spinal muscular atrophy: A proposed classification.
Proud CM, Mercuri E, Finkel RS, Kirschner J, De Vivo DC, Muntoni F, Saito K, Tizzano EF, Desguerre I, Quijano-Roy S, Benguerba K, Raju D, Faulkner E, Servais L. Proud CM, et al. Among authors: saito k. Ann Clin Transl Neurol. 2023 Nov;10(11):2155-2160. doi: 10.1002/acn3.51889. Epub 2023 Sep 10. Ann Clin Transl Neurol. 2023. PMID: 37691296 Free PMC article.
Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial.
Mercuri E, Deconinck N, Mazzone ES, Nascimento A, Oskoui M, Saito K, Vuillerot C, Baranello G, Boespflug-Tanguy O, Goemans N, Kirschner J, Kostera-Pruszczyk A, Servais L, Gerber M, Gorni K, Khwaja O, Kletzl H, Scalco RS, Staunton H, Yeung WY, Martin C, Fontoura P, Day JW; SUNFISH Study Group. Mercuri E, et al. Among authors: saito k. Lancet Neurol. 2022 Jan;21(1):42-52. doi: 10.1016/S1474-4422(21)00367-7. Lancet Neurol. 2022. PMID: 34942136
Natural history of Type 1 spinal muscular atrophy: a retrospective, global, multicenter study.
Cances C, Vlodavets D, Comi GP, Masson R, Mazurkiewicz-Bełdzińska M, Saito K, Zanoteli E, Dodman A, El-Khairi M, Gorni K, Gravestock I, Hoffart J, Scalco RS, Darras BT; ANCHOVY Working Group. Cances C, et al. Among authors: saito k. Orphanet J Rare Dis. 2022 Jul 29;17(1):300. doi: 10.1186/s13023-022-02455-x. Orphanet J Rare Dis. 2022. PMID: 35906608 Free PMC article.
Ido2 Deficiency Exacerbates Motor Impairment and Reduces Aryl Hydrocarbon Receptor Activity through Decreased Kynurenine in a Chronic Demyelinating Mouse Model.
Kunisawa K, Hara M, Yoshidomi K, Kon Y, Yamamoto Y, Fujigaki S, Wulaer B, Kosuge A, Tanabe M, Saitoh S, Takahashi K, Saito K, Nabeshima T, Mouri A. Kunisawa K, et al. Among authors: saito k. Mol Neurobiol. 2024 Jun 3. doi: 10.1007/s12035-024-04263-9. Online ahead of print. Mol Neurobiol. 2024. PMID: 38829509
8,929 results