Use of inhibitors of porphobilinogen deaminase in the treatment of congenital erythropoietic porphyria
AssigneeCENTRO DE INVESTIGACION COOPERATIVA EN BIOCIENCIAS - CIC bioGUNE
InventorsOscar Millet Aguilar-Galindo, Ana Laín Torre
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Abstract
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).
Claims16
1-7. (canceled)
A method for the prevention and/or treatment of congenital erythropoietic porphyria (CEP) in a subject in need thereof comprising the administration to said subject of a therapeutically effective amount of a compound of formula (I)
- wherein
- R1 and R2 are independently selected from the group consisting of H and C1-C6 alkyl, or R1 and R2 are bound to one another forming an optionally substituted fused benzene ring,
- R3 is selected from the group consisting of H, C1-C6 alkyl and
- R4 is selected from the group consisting of H and C1-C6 alkyl, or R4 represents
Dependencies are inferred from the claim text.