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USE OF INHIBITORS OF PORPHOBILINOGEN DEAMINASE IN THE TREATMENT OF CONGENITAL ERYTHROPOIETIC PORPHYRIA
USE OF INHIBITORS OF PORPHOBILINOGEN DEAMINASE IN THE TREATMENT OF CONGENITAL ERYTHROPOIETIC PORPHYRIA
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机译:胆红素脱氨酶抑制剂在先天性红血球性卟啉症的治疗中的应用
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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).
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