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Targeted Therapies for Leigh Syndrome: Systematic Review and Steps Towards a ‘Treatabolome’
BACKGROUND: Leigh syndrome (LS) is the most frequent paediatric clinical presentation of mitochondrial disease. The clinical phenotype of LS is highly heterogeneous. Though historically the treatment for LS is largely supportive, new treatments are on the horizon. Due to the rarity of LS, large-scal...
| Autores principales: | , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
IOS Press
2021
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8673543/ https://www.ncbi.nlm.nih.gov/pubmed/34308912 http://dx.doi.org/10.3233/JND-210715 |