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.