Therapeutic Areas - Acute Hepatic Porphyria (AHP)
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Acute Hepatic Porphyria (AHP)
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Acute Hepatic Porphyria (AHP) is a group of rare genetic diseases caused by heme biosynthesis dysregulation in the liver, leading to accumulation of neurotoxic intermediates such as aminolevulinic acid (ALA) and porphobilinogen (PBG). AHP presents with acute, potentially life-threatening attacks, and may also cause chronic symptoms that negatively impact patient functioning and quality of life.1,2
Resources
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Porphyria Burden Assessment Tool
The porphyria burden assessment (PBA) is a digitally accessible patient questionnaire intended to capture patients’ lived experiences of acute hepatic porphyria (AHP), focused on the chronic symptom burden associated with the condition and the impact of AHP on daily life.
References:
- Moghe A, Dickey A, Erwin A, et al. Mol Genet Metab. 2023;140(3):107670.
- Simon A, Pompilus F, Querbes W, et al. Patient. 2018;11(5):527-537.
