Indication

Acute intermittent porphyria

Acute intermittent porphyria is a rare, autosomal dominant metabolic disorder caused by a deficiency in the enzyme hydroxymethylbilane synthase within the heme biosynthesis pathway. Read more about Acute intermittent porphyria

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About Acute intermittent porphyria.

Acute intermittent porphyria is a rare, autosomal dominant metabolic disorder caused by a deficiency in the enzyme hydroxymethylbilane synthase within the heme biosynthesis pathway.

It predominantly affects adults, with symptomatic episodes occurring most frequently in females of reproductive age.

Pathophysiologically, hepatic upregulation of heme production leads to the toxic systemic accumulation of the neurotoxic porphyrin precursors delta-aminolevulinic acid and porphobilinogen.

The condition is clinically characterized by episodic, potentially life-threatening neurovisceral attacks that typically present with severe abdominal pain, nausea, autonomic dysfunction, peripheral neuropathy, and central neuropsychiatric disturbances, notably occurring without cutaneous lesions.

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