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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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Detalles Bibliográficos
Autores principales:	Stockler‐Ipsiroglu, Sylvia, Potter, Beth K., Yuskiv, Nataliya, Tingley, Kylie, Patterson, Marc, van Karnebeek, Clara
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley & Sons, Inc. 2020
Materias:	Review Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7891579/ https://www.ncbi.nlm.nih.gov/pubmed/32944978 http://dx.doi.org/10.1002/jimd.12315

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