Sapropterin dihydrochloride (Kuvan)
Phenylalanine hydroxylase (PAH) deficiency, traditionally known as phenylketonuria (PKU), is an autosomal recessive inborn error of metabolism caused by mutations in the gene encoding PAH, the enzyme that converts phenylalanine (Phe) to tyrosine. If untreated, Phe accumulates to toxic levels in the...
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| Format: | eBook |
| Language: | English |
| Published: |
Ottawa (ON)
CADTH
September 2017, 2017
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| Series: | Common drug review clinical review report
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| Online Access: | |
| Collection: | National Center for Biotechnology Information - Collection details see MPG.ReNa |
Table of Contents:
- Includes bibliographical references