Mitochondrial Myopathies

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Karaa, A., Bertini, E., Carelli, V., Cohen, B., Ennes, G. M., Falk, M. J., Goldstein, A., Gorman, G., Haas, R., Hirano, M., Klopstock, T., Koenig, M. K., Kornblum, C., Lamperti, C., Lehman, A., Longo, N., Molnar, M. J., Parikh, S., Phan, H., Pitceathly, R. D. S., … MMPOWER-3 Trial Investigators (2024). Genotype-specific effects of elamipretide in patients with primary mitochondrial myopathy: a post hoc analysis of the MMPOWER-3 trial. Orphanet journal of rare diseases, 19(1), 431. https://doi.org/10.1186/s13023-024-03421-5

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Karaa, A., Bertini, E., Carelli, V., Cohen, B. H., Enns, G. M., Falk, M. J., Goldstein, A., Gorman, G. S., Haas, R., Hirano, M., Klopstock, T., Koenig, M. K., Kornblum, C., Lamperti, C., Lehman, A., Longo, N., Molnar, M. J., Parikh, S., Phan, H., … Alessina, C. (2023). Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy. Neurology, 101(3). https://doi.org/10.1212/wnl.0000000000207402
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Berardo, A., Domínguez-González, C., Engelstad, K., & Hirano, M. (2022). Advances in Thymidine Kinase 2 Deficiency: Clinical Aspects, Translational Progress, and Emerging Therapies. Journal of Neuromuscular Diseases, 9(2), 225–235. https://doi.org/10.3233/jnd-210786
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