Unlocking Hope for Hurler-Scheie: 4 Therapies Steering the Next Wave of MPS I-B Care
Mucopolysaccharidosis Type I (MPS I) represents one of the most challenging genetic disorders, affecting approximately 1 in 100,000 births worldwide. This lysosomal storage disease occurs when the body lacks sufficient alpha-L-iduronidase enzyme, leading to the accumulation of glycosaminoglycans in various tissues and organs. While current treatments like enzyme replacement...
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