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Therapeutics in paediatric genetic diseases: current and future landscape
There are more than 7,000 paediatric genetic diseases (PGDs) but less than 5% have treatment options. Treatment strategies targeting different levels of the biological process of the disease have led to optimal health outcomes in a subset of patients with PGDs, where treatment is available. In the p...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Wolters Kluwer - Medknow
2023
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9979794/ https://www.ncbi.nlm.nih.gov/pubmed/36722512 http://dx.doi.org/10.4103/singaporemedj.SMJ-2021-376 |