RA 5557 is a brain-penetrant, selective, oral UGT8 inhibitor, inhibits galactosylceramide biosynthesis.
RA 5557 reduced pathologically elevated psychosine concentrations (72-86%) in the midbrain and cerebral cortex in twitcher mice.
RA 5557 decreased galactosylceramides by about 70% in midbrain and cerebral cortex in mutant and wild type animals.
RA 5557 significantly decreased several characteristic inflammatory response markers without causing apparent toxicity to myelin-producing cells in wild type and mutant mice.
RA 5557 decreases in vivo galactosphingolipid biosynthesis in murine Krabbe disease.
