Biogen and Alectos Therapeutics Announce License and Collaboration Agreement for AL01811, a Novel GBA2 Inhibitor for the Potential Treatment of Parkinson’s Disease
AL01811 is a preclinical selective GBA2 inhibitor with first-in-class potential [...]
Cytosolic Glucosylceramide Regulates Endolysosomal Function in Niemann-Pick type C Disease
Niemann-Pick type C disease (NPCD) is a neurodegenerative disease associated [...]
N-Butyldeoxynojirimycin Delays Motor Deficits, Cerebellar Microgliosis, and Purkinje Cell Loss in a Mouse Model of Mucolipidosis type IV
Mucolipidosis type IV (MLIV) is a lysosomal storage disease exhibiting [...]
Reducing GBA2 Activity Ameliorates Neuropathology in Niemann-Pick Type C Mice
The enzyme glucocerebrosidase (GBA) hydrolyses glucosylceramide (GlcCer) in lysosomes. Markedly [...]
Glucocerebrosidase 2 Gene Deletion Rescues Type 1 Gaucher Disease
The inherited deficiency of the lysosomal glucocerebrosidase (GBA) due to [...]