Emerging Paradigms in the Sandhoff Disease / GM2-gangliosidosis Type II Therapeutic Market
Sandhoff Disease also referred to as GM2-gangliosidosis Type ll is an autosomal recessive genetic lipid storage disorder that is caused by an abnormal gene for the beta subunit of the hexosaminidase B enzyme. The dysfunction of this gene causes accumulation of lipids called GM2 gangliosides in the...