Thelansis

GM2 gangliosidoses are three related genetic disorders due to a deficiency of the beta-hexosaminidase enzyme, which catalyses the biodegradation of fatty acid derivatives known as gangliosides in lysosomes. Tay-Sachs disease is the most common and involves a defect in the HEXA gene for beta-hexosaminidase A (in particular, the alpha subunit). ·        Thelansis captures epidemiology on incidence, prevalence, diagnosed, and treated prevalence for all forms of GM2 such as Tay-Sachs disease, Sandhoff disease, and AB variant. ·        The prevalence of Tay-Sachs disease is The prevalence of infection is 1 case per 320 000 live births. The AB variant is scarce; only a few cases have been reported worldwide. Based on a few sources, the incidence of Sandhoff disease is approximately 1 out of every 180,000 live births worldwide. ·        A Phase II drug candidate leads pipeline sensation for GM-2 gangliosi...