A new class of drugs treats genetic disease by stopping waste before it forms
A breakthrough drug is transforming genetic medicine by crossing the blood-brain barrier to stop toxic waste from forming in cells, offering new hope for treating rare metabolic and neurological disorders.
Traditional medicine often replaces missing enzymes, but a new class of drugs called substrate reduction therapy stops cellular waste before it ever begins. The drug venglustat targets the enzyme responsible for fat buildup in Gaucher disease, significantly improving neurological symptoms like ataxia and cognition.
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