A new key to fight rare childhood disease

A research team led by biochemist Scott Garman at the University of Massachusetts Amherst has for the first time determined the mechanism of one of the cell's 'recycling' enzymes, human alpha-galactosidase or alpha-GAL, as it breaks down substances in the lysosome, the cell's recycling centre. The work promises to aid treatment of a rare childhood metabolic disorder, Fabry disease. Patients may survive to adulthood but have compromised kidney function or heart disease, for example, due to lipid buildup in blood vessels, tissues and organs...

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