P03 Leflunomide treatment for inflammatory bowel disease and intestinal failure caused by TTC7A deficiency
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DOI: 10.1136/archdischild-2023-nppg.3
Abstract: TTC7A deficiency Ultra-rare autosomal-recessive variants in tetratricopeptide repeat domain 7A gene (TTC7A) have been discovered in patients presenting with severe intestinal disease. Mutations in the TTC7A gene cause intestinal epithelial and immune defects resulting in… read more here.
Keywords: leflunomide; ttc7a deficiency; leflunomide treatment; gastroenterology ... See more keywords