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Porphyria, Acute Intermittent
An autosomal dominant porphyria that is due to a deficiency of HYDROXYMETHYLBILANE SYNTHASE in the LIVER, the third enzyme in the 8-enzyme biosynthetic pathway of HEME. Clinical features are recurrent and life-threatening neurologic disturbances, ABDOMINAL PAIN, and elevated level of AMINOLEVULINIC ACID and PORPHOBILINOGEN in the urine.
Drugs by status
Approved for this indication (3)
Phase 2 trials (1)
Organizations
Organization Involved with Phase 3 Indications (1)
Organization Involved with Phase 2 Indications (3)
Organization Involved with Phase 1 Indications (7)