ATL-002: Tyrosinemia type I
Category
Drugs National Companies
Register type
Biotech Sanitaria
Company name
Product / Service
ATL-002: Tyrosinemia type I
Type
Biodrugs/ Drugs
Therapeutic area
Enfermedades genéticas y raras
Properties/Indication
Mutations in the FAH gene cause hereditary tyrosinemia type I, an inborn error of metabolism marked by the accumulation of tyrosine, methionine, tyrosine metabolites, and succinylacetone in blood and urine. We are developing pharmacological chaperones for this disease. This project is at the hit identification phase of discovery.
Development phase
R&D
Phases developed in Spain
R&D
Research location
España