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phenylketonuria

File:L-Phenylalanin_-_L-Phenylalanine.svg · Wikimedia Commons · See Wikimedia Commons

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phenylketonuria

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Also known as PKU, phenylalaninemia, Følling's disease, phenylketonurias

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Key facts

Medical condition (new).name
Phenylketonuria
Medical condition (new).image
L-Phenylalanin - L-Phenylalanine.svg
Medical condition (new).caption
Phenylalanine
Medical condition (new).synonyms
Phenylalanine hydroxylase deficiency, PAH deficiency, Følling disease
Medical condition (new).field
Medical genetics, pediatrics, dietetics
Medical condition (new).symptoms
Without treatment: intellectual disability, seizures, hyperactivity, psychiatric problems, musty odor
Medical condition (new).onset
At birth
Medical condition (new).onset_always
At birth
Medical condition (new).duration
Lifelong
Medical condition (new).types
Classic, variant
Medical condition (new).causes
Genetic (autosomal recessive)
Medical condition (new).diagnosis
Newborn screening programs in many countries
Medical condition (new).treatment
Diet low in foods that contain phenylalanine; special supplements
Medical condition (new).medication
Sapropterin dihydrochloride, pegvaliase
Medical condition (new).prognosis
Normal health with treatment
Medical condition (new).frequency
~1 in 12,000 newborns

via Wikipedia infobox

Research

9,270 papers

via PubMed

~25 min read

Encyclopedic overview

19 sections
Contents
  • Signs and symptoms
  • Genetics
  • Pathophysiology
  • Classical PKU
  • Tetrahydrobiopterin-deficient hyperphenylalaninemia
  • Metabolic pathways
  • Screening
  • Treatment
  • Diet
  • Nutritional supplements
  • Enzyme substitutes
  • Mothers
  • Epidemiology
  • History
  • Etymology and pronunciation
  • Research
  • See also
  • References
  • External links

Phenylketonuria (PKU) is an inborn error of metabolism that results in decreased metabolism of the amino acid phenylalanine. Untreated PKU can lead to intellectual disability, seizures, behavioral problems, and mental disorders. It may also result in a musty smell and lighter skin. A baby born to a mother who has poorly treated PKU may have heart problems, a small head, and low birth weight.

Phenylketonuria is an inherited genetic disorder. It is caused by mutations in the PAH gene, which can result in inefficient or nonfunctional phenylalanine hydroxylase, an enzyme responsible for the metabolism of excess phenylalanine. This results in the buildup of dietary phenylalanine to potentially toxic levels. It is autosomal recessive, meaning that both copies of the gene must be mutated for the condition to develop. The two main types are classic PKU and variant PKU, depending on whether any enzyme function remains. Those with one copy of a mutated gene typically do not have symptoms. Many countries have newborn screening programs for the disease.

Excerpted from Wikipedia’s “phenylketonuria” article, available under the CC BY-SA 4.0 licence.

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