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. (lysogene.com)
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. (lysogene.com)
Genzyme has long been known for our expertise in the class of rare genetic diseases known as lysosomal storage disorders (LSDs). (sciencemag.org)