Use of inhibitors of porphobilinogen deaminase in the treatment of congenital erythropoietic porphyria

摘要

The invention relates to the use of a compound of general formula (I), wherein R¹ and R² are independently H or C₁-C₆ alkyl, or R¹ and R² are bound to one another forming an optionally substituted fused benzene ring, R³ is H, C₁-C₆ alkyl or —CH₂— CH(NH₂)—COOH and R⁴ is H, C₁-C₆ alkyl, or R⁴ represents (II) or (III) in the preparation of a medicinal product for treating and/or preventing congenital erythropoietic porphyria (CEP).;