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Morquio A syndrome (mucopolysaccharidosis IVA [MPS IVA]) is a lysosomal storage disorder caused by deficiency of the enzyme N-acetylgalactosamine-6-sulfatase, which is required to degrade the glycosaminoglycan keratan sulfate. Morquio A is associated with extensive morbidity and early mortality. Elosulfase alfa is an enzyme replacement therapy that provides a treatment option for patients with Morquio...
Lysosomal storage diseases arise because of genetic mutations that result in nonfunctioning or dysfunctional lysosomal enzymes responsible for breaking down molecules such as glycosaminoglycans or glycogen. Many of these storage diseases, such as the mucopolysaccharidosis (MPS) disorders and Pompe disease, can now be treated with infusion therapies to replace the dysfunctional protein with active...
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