European Society of Human Genetics Conference (ESHG), 2025
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Survival, functional outcomes and safety in patients with thymidine kinase 2 deficiency (TK2d) and an age of TK2d symptom onset ≤12 years who received pyrimidine nucleos(t)ide therapy
Caterina Garone, Cristina Domínguez-González, Richard Haas, et al.
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