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309 results

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Safety and efficacy of leriglitazone for preventing disease progression in men with adrenomyeloneuropathy (ADVANCE): a randomised, double-blind, multi-centre, placebo-controlled phase 2-3 trial.
Köhler W, Engelen M, Eichler F, Lachmann R, Fatemi A, Sampson J, Salsano E, Gamez J, Molnar MJ, Pascual S, Rovira M, Vilà A, Pina G, Martín-Ugarte I, Mantilla A, Pizcueta P, Rodríguez-Pascau L, Traver E, Vilalta A, Pascual M, Martinell M, Meya U, Mochel F; ADVANCE Study Group. Köhler W, et al. Among authors: fatemi a. Lancet Neurol. 2023 Feb;22(2):127-136. doi: 10.1016/S1474-4422(22)00495-1. Lancet Neurol. 2023. PMID: 36681445 Clinical Trial.
A Phase 1 Study of Oral Vitamin D3 in Boys and Young Men With X-Linked Adrenoleukodystrophy.
Van Haren KP, Cunanan K, Awani A, Gu M, Peña D, Chromik LC, Považan M, Rossi NC, Goodman J, Sundaram V, Winterbottom J, Raymond GV, Cowan T, Enns GM, Waubant E, Steinman L, Barker PB, Spielman D, Fatemi A. Van Haren KP, et al. Among authors: fatemi a. Neurol Genet. 2023 Mar 31;9(2):e200061. doi: 10.1212/NXG.0000000000200061. eCollection 2023 Apr. Neurol Genet. 2023. PMID: 37090939 Free PMC article.
Practical Approach to Longitudinal Neurologic Care of Adults With X-Linked Adrenoleukodystrophy and Adrenomyeloneuropathy.
Kornbluh AB, Baldwin A, Fatemi A, Vanderver A, Adang LA, Van Haren K, Sampson J, Eichler FS, Sadjadi R, Engelen M, Orthmann-Murphy JL. Kornbluh AB, et al. Among authors: fatemi a. Neurol Genet. 2024 Oct 3;10(5):e200192. doi: 10.1212/NXG.0000000000200192. eCollection 2024 Oct. Neurol Genet. 2024. PMID: 39372123 Free PMC article. Review.
Longitudinal natural history studies based on real-world data in rare diseases: Opportunity and a novel approach.
Adang LA, Sevagamoorthy A, Sherbini O, Fraser JL, Bonkowsky JL, Gavazzi F, D'Aiello R, Modesti NB, Yu E, Mutua S, Kotes E, Shults J, Vincent A, Emrick LT, Keller S, Van Haren KP, Woidill S, Barcelos I, Pizzino A, Schmidt JL, Eichler F, Fatemi A, Vanderver A. Adang LA, et al. Among authors: fatemi a. Mol Genet Metab. 2024 May;142(1):108453. doi: 10.1016/j.ymgme.2024.108453. Epub 2024 Mar 18. Mol Genet Metab. 2024. PMID: 38522179 Review.
309 results