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Drug treatment of inborn errors of metabolism: a systematic review.


ABSTRACT:

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 sources but tend to vary among these sources. Moreover, the published dosages are not usually supported by the level of existing evidence, and they are commonly based on personal experience.

Methods

A literature search was conducted to identify key material published in English in relation to the dosages of medicines used for specific IEM. Textbooks, peer reviewed articles, papers and other journal items were identified. The PubMed and Embase databases were searched for material p

SUBMITTER: Alfadhel M 

PROVIDER: S-EPMC3693126 | biostudies-literature | 2013 Jun

REPOSITORIES: biostudies-literature