Lamzede Gets Approved for Rare Disease Alpha-Mannosidosis
XTalks
FEBRUARY 27, 2023
A lack of this enzyme results in a toxic build-up of mannose-containing sugars in the cells of the body, including the central nervous system (CNS). The disease usually clearly presents between birth and childhood, though it is often not recognized and patients can be undiagnosed and untreated for long periods.
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