Exogenous Galactosylceramide as Potential Treatment for CLN3 Disease.

Sally El-SittJihane SoueidKatia MaaloufNadine MakhoulJamal Al AliJoelle MakoukjiBilal AsserDaniel DaouHayat HaratiRose-Mary N Boustany

Published in: Annals of neurology (2019)

Galactosylceramide improved behavioral, neuropathological, and biochemical parameters in Cln3 Δex7/8 mice, paving the way for effective therapy for CLN3 disease and use of serum ceramide as a potential biomarker to track impact of therapies. ANN NEUROL 2019;86:729-742.

Keyphrases

type diabetes
metabolic syndrome
adipose tissue
high fat diet induced
skeletal muscle
insulin resistance
combination therapy