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David Cracc adicionou um novo artigo Health
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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David Cracc adicionou um novo artigo Health
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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