Lysosomal Storage Disorders, or LSDs, are rare genetic conditions that may not make headlines, but they affect thousands worldwide, often with life-changing consequences. LSDs happen when the body’s lysosomes, tiny cellular recycling centers, fail to break down waste materials due to missing or faulty enzymes. This leads to a buildup of harmful substances, causing damage to organs like the brain, heart, and bones. Globally, LSDs impact about 1 in 5K to 1 in 10K people, with over 70 distinct disorders identified, totaling around 700K patients worldwide.
Living with an LSD can be tough. Symptoms vary widely, from developmental delays and seizures to bone deformities ...