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Developments in evidence creation for treatments of inborn errors of metabolism
Inborn errors of metabolism (IEM) represent the first group of genetic disorders, amenable to causal therapies. In addition to traditional medical diet and cofactor treatments, new treatment strategies such as enzyme replacement and small molecule therapies, solid organ transplantation, and cell‐and...
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| Publicado no: | J Inherit Metab Dis |
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| Main Authors: | , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
John Wiley & Sons, Inc.
2020
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7891579/ https://ncbi.nlm.nih.gov/pubmed/32944978 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jimd.12315 |
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