Arylsulfatase B, also known as Galsulfase, is an enzyme replacement therapy used to treat mucopolysaccharidosis type VI (MPS VI), a rare genetic disorder. MPS VI is caused by the deficiency of arylsulfatase B, an enzyme needed to break down certain long-chain sugars in the body. Galsulfase is a form of arylsulfatase B that is designed to replace the missing enzyme. It is given as an intravenous infusion every two weeks, and works to help break down the long-chain sugars, which can help improve a variety of symptoms caused by MPS VI. Galsulfase has been shown to help improve physical strength, mobility, and eye and ear function in people with MPS VI.
Galsulfase is a recombinant human enzyme used as replacement enzyme therapy for the treatment of adults and children with Mucopolysaccharidosis VI (MPS VI), a rare genetic disorder caused by a deficiency of a lysosomal enzyme [1]. It is sold under the brand name Naglazyme [2].
MPS VI, also known as Maroteaux-Lamy syndrome, is a lysosomal storage disorder that affects many parts of the body. It is caused by a deficiency of the enzyme N-acetylgalactosamine 4-sulfatase, which is needed to break down glycosaminoglycans (GAGs) in the body. When MPS VI is present, GAGs build up in cells, tissues, and organs throughout the body, leading to progressive damage.
Galsulfase works by replacing the missing enzyme in people with MPS VI. This helps to break down GAGs and slow down the progression of the disease. Galsulfase is given by intravenous (IV) infusion. [1]
Here are some important things to know about galsulfase:
- It is not a cure for MPS VI, but it can help to improve symptoms and slow down the progression of the disease.
- Galsulfase can cause serious side effects, including allergic reactions, infusion reactions, and worsening of MPS VI symptoms.
- Galsulfase is a very expensive medication.
If you are considering treatment with galsulfase, it is important to talk to your doctor about the risks and benefits of this medication.