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Lysosomal Storage Disorders: CI Insights into Rare Disease Innovation and Market Trends
Lysosomal storage diseases (LSDs) are a group of more than 70 rare inherited metabolic disorders caused by the malfunction of lysosomal enzymes. These enzymes are responsible for breaking down various substances in cells. When they fail, harmful materials accumulate in lysosomes, leading to cellular dysfunction, progressive organ damage, and, in many cases, life-threatening complications....
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