[EN] CERAMIDE GALACTOSYLTRANSFERASE INHIBITORS FOR THE TREATMENT OF DISEASE<br/>[FR] INHIBITEURS DE LA CÉRAMIDE GALACTOSYLTRANSFÉRASE POUR LE TRAITEMENT DE MALADIES
申请人:BIOMARIN PHARM INC
公开号:WO2017214505A1
公开(公告)日:2017-12-14
Described herein are compounds, methods of making such compounds, pharmaceutical compositions and medicaments containing such compounds, and methods of using such compounds to treat or prevent diseases or disorders associated with the enzyme ceramide galactosyltransferase (CGT), such as, for example, lysosomal storage diseases. Examples of lysosomal storage diseases include, for example, Krabbe disease and Metachromatic Leukodystrophy.
Diamidines of the formula ##STR1## wherein X is a propylene, isobutylene, guanidine, pyrrole, tetrazole, imidazole or substituted imidazole group; and 2-[4-(2-imidazolinyl)phenyl]-6-(2-imidazolinyl)indole, are useful in the treatment of certain protozoal infections in mammals, particularly in cattle.
Diamidines of the formula ##STR1## wherein X is a propylene, isobutylene, guanidine, pyrrole, tetrazole, imidazole or substituted imidazole group; and 2-[4-(2-imidazolinyl)phenyl]-6-(2-imidazolinyl)indole, are useful in the treatment of certain protozoal infections in mammals, particularly in cattle.