[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:WO2019104011A1
公开(公告)日:2019-05-31
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.
Rate constants of solvolysis of 3′- and 4′-substituted 1-(4-biphenylyl) ethyl chlorides were measured in 80% (v/v) aqueous acetone. The effect of substituents could be correlated excellently with the LArSR relationship.log k⁄k0=−1.56(σ0+0.84Δ\barσR+)The ρ and r+ values are smaller than those in the corresponding phenyl system under identical conditions (ρ=−4.95, r+=1.15). The difference of the r+ values