Tay-Sachs is a lysosomal storage disorder
caused by the deficiency of vital enzymes called beta-hexosaminidase A (Hex-A).
The role of Hex-A is to degrade a fatty substance or lipid called GM-2
ganglioside. In the absence of the Hex-A enzymes, GM-2 accumulates abnormally
in cells, especially in the nerve cells or neurons, of the brain.
In children,
the destructive process begins in the fetus early in pregnancy. However, a baby
with Tay-Sachs disease appears normal till six months of age when its develops
slowly. By about two years of age, most children experience recurrent seizures
and diminishing mental functioning. The infant gradually regresses, and is
eventually unable to crawl, turn over, sit or reach out. There is no specific
treatment for Tay-Sachs disease.
The drug
candidates in Tay-Sachs therapeutics pipeline include, but not limited to,
CCP-010. Some of the major companies having drugs in the Tay-Sachs therapeutics
pipeline includes Lysogene, and Castle Creek Pharmaceuticals.
The report
provides a comprehensive understanding of the pipeline activities covering all
drug candidates under various stages of development, with the detailed analysis
of pipeline and clinical trials.
Pipeline
analysis of drugs by phases includes product description and development
activities including information about clinical results, designations,
collaborations, licensing, grants, technology, and others.
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