Advancing Mucopolysaccharidosis Type I Treatment

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2025-04-29 10:43:05

Advancing Mucopolysaccharidosis Type I Treatment — 4 Potential Therapies That Could Change the Landscape

Mucopolysaccharidosis Type I (MPS I) is a rare and complex genetic disorder, resulting from a deficiency of the enzyme alpha-L-iduronidase, which leads to the accumulation of glycosaminoglycans (GAGs) in cells. This buildup can severely affect various organs, including the heart, liver, and brain. While existing treatments like enzyme replacement therapy (ERT) and hematopoietic stem cell...

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