Mucopolysaccharidosis Type I – Pipeline Insight, 2025:...

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2025-07-21 13:44:54 - Перевод -

Mucopolysaccharidosis Type I – Pipeline Insight, 2025: Exploring Emerging Therapies and Future Treatment Potential

Mucopolysaccharidosis Type I is a rare genetic lysosomal storage disease caused by the lack of the alpha-L-iduronidase enzyme. This enzyme deficiency results in the buildup of glycosaminoglycans (GAGs) in tissues and organs, leading to a range of symptoms that vary in intensity—from mild to severe. The condition is categorized into three forms: Hurler syndrome, Hurler-Scheie syndrome, and...

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