Maroteaux Lamy Syndrome is Amazingly Expensive to Treat

If you have Maroteaux Lamy syndrome, there’s good news and not-so-good news.

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On the one hand, an understanding of this rare genetic disease has been reached by the medical community. Also, pharma companies have been busily trying to come up with treatments apart from the enzyme replacement therapy Naglazyme® (galsulfase), which was approved by the FDA in 2005.

On the other hand, as welcome as it is on the market, Naglazyme remains one of the most expensive treatments in the United States.

Maroteaux-Lamy, an autosomal disorder, is characterized by a complete or partial malfunctioning of an enzyme called arylsulfatase B. This causes a buildup of glycosaminoglycans (a fancy word for complex carbohydrates) and can affect multiple organs in the body. Some people with the disorder can remain symptom-free throughout their lives, while others are not as fortunate.

Maroteaux Lamy Syndrome Symptoms

Symptoms include:

  • Clouding of the corneas
  • Coarse facial features
  • Short stature
  • Liver and spleen enlargement

Complications, in the most severe cases, can be life-threatening. For many, joint pain and skeletal malformations are another part of their rare disease.

And Now, Back to Those Pesky $$…

Because of the high cost of Naglazyme, many people have difficulty paying for it. If you are one of these patients, the Partnership for Prescription Assistance may be able to help. Their services are free of charge, and they may be able to help you find sources to help you accesses your needed medications.

You can also contact BioMarin, the pharmaceutical company that makes Naglazyme and request information on their Compassionate Use policy.

Did you find help paying for expensive medicines? Share your success stories and resources in the comments below!

Erica Zahn

Erica Zahn

Erica Zahn is passionate about raising awareness of rare diseases and disorders and helping people connect with the resources that may ease their journey. Erica has been a caregiver, and is a patient, herself, so she completely relates to the rare disease community--on a deeply personal level.

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