Indication

Alcaptonuria

Alcaptonuria (also spelled alkaptonuria) is a rare autosomal recessive inborn error of metabolism caused by a deficiency in the hepatic enzyme homogentisate 1,2-dioxygenase, which disrupts the normal catabolism of phenylalanine and tyrosine. Read more about Alcaptonuria

9 drugs 7 manufacturers

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Drugs that treat it

Common Alcaptonuria therapies.

Showing 1–9 of 9 drugs

ORFADIN (nitisinone)

DRUGS TO TREAT HEREDITARY TYROSINEMIA·HEREDITARY TYROSINEMIA TYPE I·+1
BIOVITRUM

ORFADIN (nitisinone)

DRUGS TO TREAT HEREDITARY TYROSINEMIA·HEREDITARY TYROSINEMIA TYPE I·+1
BIOVITRUM

NITYR (nitisinone)

DRUGS TO TREAT HEREDITARY TYROSINEMIA·HEREDITARY TYROSINEMIA TYPE I·+1
CYCLE PHARMACEU

NITISINONE

DRUGS TO TREAT HEREDITARY TYROSINEMIA·HEREDITARY TYROSINEMIA TYPE I·+1
PAR HEALTH USA,

NITISINONE

DRUGS TO TREAT HEREDITARY TYROSINEMIA·HEREDITARY TYROSINEMIA TYPE I·+1
TORRENT PHARMAC

NITISINONE

DRUGS TO TREAT HEREDITARY TYROSINEMIA·HEREDITARY TYROSINEMIA TYPE I·+1
BRYANT RANCH PR

NITISINONE

DRUGS TO TREAT HEREDITARY TYROSINEMIA·HEREDITARY TYROSINEMIA TYPE I·+1
ANALOG PHARMA,

NITISINONE

DRUGS TO TREAT HEREDITARY TYROSINEMIA·HEREDITARY TYROSINEMIA TYPE I·+1
ETON PHARMACEUT

HARLIKU (nitisinone)

DRUGS TO TREAT HEREDITARY TYROSINEMIA·HEREDITARY TYROSINEMIA TYPE I·+1
CYCLE PHARMACEU
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About Alcaptonuria.

Alcaptonuria (also spelled alkaptonuria) is a rare autosomal recessive inborn error of metabolism caused by a deficiency in the hepatic enzyme homogentisate 1,2-dioxygenase, which disrupts the normal catabolism of phenylalanine and tyrosine.

This enzymatic defect leads to the systemic accumulation and urinary excretion of homogentisic acid, which oxidizes into dark benzoquinone acetic acid polymers.

Clinically, it manifests in infancy with urine that darkens upon air exposure, followed by adult-onset ochronosis—characterized by bluish-black pigment deposition in connective tissues.

Over time, this pigment deposition leads to severe, early-onset ochronotic osteoarthropathy of the spine and weight-bearing joints, as well as complications such as heart valve calcification, nephrolithiasis, and tendon rupture.

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