ICD E80ORPHA:79276AHP

Porphyria

Porphyria refers to a group of rare disorders caused by enzyme deficiencies in the heme biosynthesis pathway, affecting the nervous system or skin. Acute porphyrias cause potentially life-threatening attacks of abdominal pain and neurological symptoms. A new RNAi therapy (givosiran) has transformed prevention of attacks.

244
Articles
65
Trials (6 AU)
Updated
25 March 2026
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Common Questions

What is Porphyria?

Porphyria refers to a group of rare disorders caused by enzyme deficiencies in the heme biosynthesis pathway, affecting the nervous system or skin. Acute porphyrias cause potentially life-threatening attacks of abdominal pain and neurological symptoms. A new RNAi therapy (givosiran) has transformed prevention of attacks.

How many clinical trials are available for Porphyria?

RareWays currently indexes 65 clinical trials for Porphyria, of which 8 are actively recruiting. Trial availability changes as new studies are registered — check the trials tab for current status.

Where does the research data for Porphyria come from?

RareWays aggregates research from PubMed, Europe PMC, OpenAlex, and ClinicalTrials.gov. Data is updated regularly by Rocky, RareWays' automated research engine. All articles and trials link directly to their original sources.

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This information is for general awareness only.

For guidance specific to your situation, please speak with your healthcare team.