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What is Mepsevii used to treat?

Answers (1)

Official Answer by Drugs.com 23 Jan 2018

Mepsevii (vestronidase alfa-vjbk) from Ultragenyx Pharmaceuticals is used to treat pediatric and adult patients with an inherited lysosomal storage disorder called mucopolysaccharidosis type VII (MPS VII), also known as Sly Syndrome. In this disorder, a deficiency of the enzyme beta-glucuronidase leads to a build-up of carbohydrates in cells of the joints, brain, spinal cord, heart, and other organs. Mepsevii replaces this missing enzyme in the body to aid in the metabolism of the carbohydrates.

What is mucopolysaccharidosis type VII (MPS VII)?

Signs and symptoms of mucopolysaccharidosis type VII (MPS VII) include short stature due to growth inhibition, skeletal structure abnormalities, and some degree of intellectual impairment in certain patients, among others. MPS VII is very rare, occurring in roughly one in 250,000 births.

What clinical studies were done with Mepsevii?

Safety and effectiveness of Mepsevii was shown in a clinical study of 23 patients ranging from 5 months to 25 years. Positive results with Mepsevii were seen in patients with the six-minute walk test and improvement in pulmonary function. The most common side effects include infusion site reactions, diarrhea, rash and anaphylaxis (a severe allergic reaction).

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