Animal models of methylmalonic acidemia: insights and challenges
Abstract Methylmalonic acidemia (MMA) is a rare genetic disorder caused by disruptions in the metabolism of methylmalonic acid, resulting in severe neurological and systemic complications. Animal models have become indispensable in advancing our understanding of MMA pathogenesis, evaluating treatmen...
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| Autori principali: | , , , , , |
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| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
BMC
2025-11-01
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| Serie: | Orphanet Journal of Rare Diseases |
| Soggetti: | |
| Accesso online: | https://doi.org/10.1186/s13023-025-04101-8 |
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