Alkaptonuria (Black Urine Disease) Medication

Updated: Mar 18, 2021
  • Author: Karl S Roth, MD; Chief Editor: Maria Descartes, MD  more...
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Medication

Medication Summary

Nitisinone is now known to be useful in managing alkaptonuria. Vitamin C, as much as 1 g/d, is also recommended for older children and adults. [10, 11, 22]

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Vitamins

Class Summary

Organic substances required by the body in small amounts for various metabolic processes.

Ascorbic acid (Cecon, Cevalin, Cevi-Bid, Ce-Vi-Sol)

The mild antioxidant nature of ascorbic acid helps to retard the process of conversion of homogentisate to the polymeric material that is deposited in cartilaginous tissues.

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Enzyme inhibitors

Class Summary

Nitisinone, a tyrosine degradation inhibitor, has been used experimentally.

Nitisinone (Orfadin)

This compound has, until recently seen very restricted use in experimental treatment for alkaptonuria. Tyrosine degradation inhibitor. Inhibits 4-hydroxyphenylpyruvate dioxygenase, which mediates formation of homogentisic acid. Its clinical use has now been shown to be safe and effective.

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