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