What is NAGLAZYME used for?
Naglazyme is used to treat patients who have mucopolysaccharidosis VI (MPS VI or Maroteaux-Lamy syndrome). This disease is caused by the lack of an enzyme called N-acetylgalactosamine 4-sulfatase, which is needed to break down substances in the body called glycosaminoglycans (GAGs).
How much does NAGLAZYME cost?
The cost for Naglazyme intravenous solution (1 mg/mL) is around $2,194 for a supply of 5 milliliters, depending on the pharmacy you visit. Prices are for cash paying customers only and are not valid with insurance plans.
What is NAGLAZYME made of?
NAGLAZYME is a formulation of galsulfase, which is a purified human enzyme that is produced by recombinant DNA technology in a Chinese hamster ovary cell line. Galsulfase (glycosaminoglycan N–acetylgalactosamine 4-sulfatase, EC 3.1.
How to get NAGLAZYME?
NAGLAZYME is given by infusion. This means that once a week, you will go to a hospital or infusion center. For some people, getting infusions at home may also be an option. During your infusion, a doctor or nurse will give you NAGLAZYME through an intravenous (IV) line.
How much does Cinryze cost?
The cost for Cinryze intravenous powder for injection 500 units is around $2,976 for a supply of 1 powder for injection, depending on the pharmacy you visit. Prices are for cash paying customers only and are not valid with insurance plans.
How much does Myozyme cost?
Myozyme, which inspired the Harrison Ford movie Extraordinary Measures, costs up to $100,000 for a child. But according to Genzyme, the average cost of adult treatment is $300,000 per year. (Because of recent manufacturing snafus, most adults with the disease are currently getting an experimental version for free.)
What is MPS VI?
Mucopolysaccharidosis type VI (MPS VI), also known as Maroteaux-Lamy syndrome, is a progressive condition that causes many tissues and organs to enlarge, become inflamed or scarred, and eventually waste away (atrophy). Skeletal abnormalities are also common in this condition.
What is Maroteaux Lamy Syndrome?
What is CINRYZE used to treat?
CINRYZE was the first C1 esterase inhibitor (C1-INH) approved by the FDA to help prevent swelling attacks in children (6 years of age and older), teenagers and adults. Available since 2008, CINRYZE has successfully been used to help reduce the frequency, severity, and length of attacks in adolescent and adult patients.
When was CINRYZE approved?
The human plasma-derived C1 esterase inhibitor (Cinryze™), distributed by Lev Pharmaceuticals, was approved in October 2008 for the prevention of HAE attacks based on the results of a phase III clinical trial.
Is Pompe disease curable?
Unfortunately, no cure exists. However, Pompe disease has benefited from the introduction of enzyme replacement therapy (ERT), which, although expensive, is a major therapeutic advance.
How do people get Pompe?
Pompe disease is inherited in an autosomal recessive pattern. Recessive genetic disorders occur when an individual inherits a non-working gene from each parent. If an individual receives one working gene and one non-working gene for the disease, the person will be a carrier for the disease.