Coverage: 2020-01-01 onwards, with up to 300 results per query from PubMed and Europe PMC and separate limits on other sources. This is a selected index, not a complete literature search.
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Tyrosinaemia Type 1 — Research Summary
Printed from RareWays (rareways.org) on 26 September 2026
For general awareness only. Not medical advice. Discuss all care options with your healthcare team.
5 Most Recent Research Articles
- 1.
Hepatocellular carcinoma in a child with hereditary tyrosinemia type I despite early NTBC therapy: When should we suspect it?
Ouassou Karima Larbi et al. — Radiology case reports (1 September 2026)
https://pubmed.ncbi.nlm.nih.gov/42293468/
- 2.
An optimized engineered bacterium for tyrosinemia type 1 therapy: A multi-species preclinical study.
Gu Peng et al. — Molecular therapy : the journal of the American Society of Gene Therapy (5 August 2026)
https://pubmed.ncbi.nlm.nih.gov/42226462/
- 3.
Engineering the gut as an auxiliary tyrosine disposal unit in hereditary tyrosinemia type 1.
De Kock Joery et al. — Molecular therapy : the journal of the American Society of Gene Therapy (5 August 2026)
https://pubmed.ncbi.nlm.nih.gov/42471012/
- 4.
CRISPR/Cas9-mediated gene correction in newborn rabbits with hereditary tyrosinemia type I
Jingke Xie et al. — 中国科学院科学数据中心 (10 July 2026)
https://doi.org/10.57841/casdc.0007460
- 5.
Multiple Hepatocellular Adenomas in a Child with Tyrosinemia Type 1 Mimicking Hepatocellular Carcinoma
Jaryung Han et al. — Convergence Hepatology (31 May 2026)
https://doi.org/10.65633/ch.2026.2.1.56
Source: RareWays research directory. Data from PubMed, Europe PMC, OpenAlex, ClinicalTrials.gov.
Always verify information with your healthcare team before making any decisions about your care.
Tyrosinaemia Type 1
Tyrosinaemia type 1 is a rare inherited condition in which the body cannot fully break down the amino acid tyrosine because an enzyme is missing. Harmful by-products build up and can damage the liver, kidneys and nerves, often from early infancy. Management includes the medicine nitisinone, a diet low in tyrosine and phenylalanine, and lifelong monitoring of liver health.
Most Recent Research
Tyrosinemia is one of the rare metabolic diseases in children. Treatment with NTBC has significantly reduced the risk of cirrhosis and therefore of neoplastic complications, including hepatocellular carcinoma, especially if treatment is started early. Close monitoring of children with this disease is necessary and must include thorough clinical, biological, and radiological investigations in the event of any suspicion of malignant complications. Liver transplantation has been the subject of much controversy in the curative treatment of the disease, as well as its major complication, hepatocellular carcinoma.
Common Questions
What is Tyrosinaemia Type 1?
Tyrosinaemia type 1 is a rare inherited condition in which the body cannot fully break down the amino acid tyrosine because an enzyme is missing. Harmful by-products build up and can damage the liver, kidneys and nerves, often from early infancy. Management includes the medicine nitisinone, a diet low in tyrosine and phenylalanine, and lifelong monitoring of liver health.
How many clinical trials are available for Tyrosinaemia Type 1?
RareWays currently indexes 1 clinical trial for Tyrosinaemia Type 1. Trial availability changes as new studies are registered — check the trials tab for current status.
Where does the research data for Tyrosinaemia Type 1 come from?
RareWays aggregates research from PubMed, Europe PMC, OpenAlex, and ClinicalTrials.gov. Data is refreshed automatically every day. All articles and trials link directly to their original sources.
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