Revolutionizing Mucopolysaccharidosis Type I Management: From...

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2025-04-29 16:53:33

Revolutionizing Mucopolysaccharidosis Type I Management: From Enzyme Therapy to Gene Therapy

Mucopolysaccharidosis type I (MPS I) is a rare metabolic disorder caused by a deficiency in the enzyme alpha-L-iduronidase, leading to the accumulation of harmful substances in cells. This condition can lead to severe complications, including developmental delays, heart problems, and joint stiffness. There are three main forms of MPS I: Hurler syndrome, Hurler-Scheie syndrome, and...

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