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Estimated prevalence: <1 in 1,000,000 (VERY_RARE).
No clinical trials have been registered for hemorrhagic disease due to alpha-1-antitrypsin Pittsburgh mutation.
2 publications have been identified in PubMed for hemorrhagic disease due to alpha-1-antitrypsin Pittsburgh mutation. Research spans Review / Meta-Analysis (50%) and Gene Therapy / Novel Therapeutics (50%).
Luís MA (2025). [PMID: 39861752](https://pubmed.ncbi.nlm.nih.gov/39861752/). *Pharmaceutics*. [Gene Therapy / Novel Therapeutics]
Fromme M (2025). [PMID: 40227077](https://pubmed.ncbi.nlm.nih.gov/40227077/). *Hepatology communications*. [Review / Meta-Analysis]
Data assembled from 3 of 12 sources · Last updated Sep 20, 2026, 5:40 PM UTC
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European rare disease database
Genetic and Rare Diseases Info Center