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Year | Number of Results |
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2019 | 1 |
2021 | 1 |
2024 | 0 |
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Therapy Prospects for Mitochondrial DNA Maintenance Disorders.
Int J Mol Sci. 2021 Jun 16;22(12):6447. doi: 10.3390/ijms22126447.
Int J Mol Sci. 2021.
PMID: 34208592
Free PMC article.
Review.
Deoxynucleoside Therapy for Thymidine Kinase 2-Deficient Myopathy.
Domínguez-González C, Madruga-Garrido M, Mavillard F, Garone C, Aguirre-Rodríguez FJ, Donati MA, Kleinsteuber K, Martí I, Martín-Hernández E, Morealejo-Aycinena JP, Munell F, Nascimento A, Kalko SG, Sardina MD, Álvarez Del Vayo C, Serrano O, Long Y, Tu Y, Levin B, Thompson JLP, Engelstad K, Uddin J, Torres-Torronteras J, Jimenez-Mallebrera C, Martí R, Paradas C, Hirano M.
Domínguez-González C, et al.
Ann Neurol. 2019 Aug;86(2):293-303. doi: 10.1002/ana.25506. Epub 2019 Jun 17.
Ann Neurol. 2019.
PMID: 31125140
Free PMC article.
METHODS: We administered deoxynucleoside monophosphates and deoxynucleoside to 16 TK2-deficient patients under a compassionate use program. RESULTS: In 5 patients with early onset and severe disease, survival and motor functions were better than historically untreated pati …
METHODS: We administered deoxynucleoside monophosphates and deoxynucleoside to 16 TK2-deficient patients under a compassionate use progra …
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