Not exact matches
The drug candidate is intended for the treatment of subjects with neuronal ceroid lipofuscinosis, aka Batten Disease, a fatal
lysosomal storage disease that primarily affects the nervous
system in children.
Both MPS IIIA and GM1 are classified as neuropathic (i.e. affecting the central nervous
system)
Lysosomal Storage Disorders, a group of approximately 50 rare inherited metabolic disorders that result from defects in lysosomal
Lysosomal Storage Disorders, a group of approximately 50 rare inherited metabolic disorders that result from defects in
lysosomal lysosomal function.
They soon concluded that the same selective
lysosomal degradation pathway was used in both
systems, even when breaking down completely different proteins.
The 4 primary intracellular pathways that contribute to the regulation of skeletal muscle proteolysis are the autophagy /
lysosomal, calpain - dependent, caspase - mediated, and ubiquitin proteasome (UP)
systems.
It's a metabolic storage disorder resulting in
lysosomal enzyme deficiency, which affects nearly every
system in the dog's body.