Substrate reduction therapy for Krabbe disease and metachromatic leukodystrophy using a novel ceramide galactosyltransferase inhibitor.

Treatment with 0.15 mg/kg S202 IP three times per week showed a trend toward reduction in the levels of CD68-positive macrophages (Fig. 5 A,B) and a marker of astrocytosis (GFAP, Fig. 5 C,D) in Twitcher mouse brain sections.

also in 53,322 other papers