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Drug treatment of inborn errors of metabolism: a systematic review

BACKGROUND: The treatment of inborn errors of metabolism (IEM) has seen significant advances over the last decade. Many medicines have been developed and the survival rates of some patients with IEM have improved. Dosages of drugs used for the treatment of various IEM can be obtained from a range of...

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Detalles Bibliográficos
Autores principales: Alfadhel, Majid, Al-Thihli, Khalid, Moubayed, Hiba, Eyaid, Wafaa, Al-Jeraisy, Majed
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BMJ Publishing Group 2013
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3693126/
https://www.ncbi.nlm.nih.gov/pubmed/23532493
http://dx.doi.org/10.1136/archdischild-2012-303131