GM1 gangliosidosis, or Landing disease, is a rare inherited
neurodegenerative lysosomal storage disorder characterized by severe cognitive and motor developmental delays resulting in the death of most patients at a very young age.
Mucopolysaccharidosis type IIIA (MPS IIIA), or Sanfilippo syndrome type A, is a rare inherited neurodegenerative
lysosomal storage disorder characterized by intractable behavioural problems and developmental regression resulting in early death.
The research establishes a new model for
studying lysosomal storage disorders and suggests that GM2 accumulation not only causes neurodegeneration but also may disrupt neurodevelopment prior to birth.
New research indicates that placental stem cell engraftment may represent an interesting new strategy to treat an enzyme deficiency associated with a lysosomal storage disorder