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Page 1
Randomized controlled trial of N-acetylcysteine therapy for RYR1-related myopathies.
Todd JJ, Lawal TA, Witherspoon JW, Chrismer IC, Razaqyar MS, Punjabi M, Elliott JS, Tounkara F, Kuo A, Shelton MO, Allen C, Cosgrove MM, Linton M, Michael D, Jain MS, Waite M, Drinkard B, Wakim PG, Dowling JJ, Bönnemann CG, Emile-Backer M, Meilleur KG. Todd JJ, et al. Among authors: emile backer m. Neurology. 2020 Mar 31;94(13):e1434-e1444. doi: 10.1212/WNL.0000000000008872. Epub 2020 Jan 15. Neurology. 2020. PMID: 31941795 Free PMC article. Clinical Trial.
Mixed methods analysis of Health-Related Quality of Life in ambulant individuals affected with RYR1-related myopathies pre-post-N-acetylcysteine therapy.
Capella-Peris C, Cosgrove MM, Chrismer IC, Emile-Backer M, Razaqyar MS, Elliott JS, Kuo A, Wakim PG, Meilleur KG. Capella-Peris C, et al. Among authors: emile backer m. Qual Life Res. 2020 Jun;29(6):1641-1653. doi: 10.1007/s11136-020-02428-2. Epub 2020 Feb 10. Qual Life Res. 2020. PMID: 32040747 Free PMC article.
Rycal S48168 (ARM210) for RYR1-related myopathies: a phase one, open-label, dose-escalation trial.
Todd JJ, Lawal TA, Chrismer IC, Kokkinis A, Grunseich C, Jain MS, Waite MR, Biancavilla V, Pocock S, Brooks K, Mendoza CJ, Norato G, Cheung K, Riekhof W, Varma P, Colina-Prisco C, Emile-Backer M, Meilleur KG, Marks AR, Webb Y, Marcantonio EE, Foley AR, Bönnemann CG, Mohassel P. Todd JJ, et al. Among authors: emile backer m. EClinicalMedicine. 2024 Jan 25;68:102433. doi: 10.1016/j.eclinm.2024.102433. eCollection 2024 Feb. EClinicalMedicine. 2024. PMID: 38318125 Free PMC article.