Mucopolysaccharidosis Type III
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Approved and Donation Ready
This fund has been developed in response to patients who have contacted PAF for help with their medication expenses and could not find help. While this fund has been fully designed and we are ready to provide needed support to these patient communities, this fund is not yet able to accept applications for assistance as we are still working to secure charitable donations that will allow us to open it.
If you, or someone you know, would like to contribute to this fund, please visit our Donors page for more information on how to provide critical support for patients in need.Fund Type
Co-Pay, Co-insurance & Deductible (medications and office visits)Maximum Award Level
- Household Income Requirements 400% or less of Federal Poverty Guideline (FPG) (adjusted for Cost of Living Index (COLI) and number in household)
- Insurance Requirements All Insurance Types
- Must reside and receive treatment in the United States.
About Mucopolysaccharidosis Type III
Mucopolysaccharidosis type III (MPS III) is a genetic disorder that makes the body unable to break down large sugar molecules called glycosaminoglycans (GAGs, formerly called mucopolysaccharides). Specifically, people with this condition are unable to break down a GAG called heparan sulfate. Affected individuals can have severe neurological symptoms, including progressive dementia, aggressive behavior, hyperactivity, seizures, deafness, loss of vision, and an inability to sleep for more than a few hours at a time.
These materials remain stored in the body’s cells, causing progressive damage. There have been four different enzyme deficiencies found, described as type A, B, C or D. MPS III is the most common form of mucopolysaccharidosis, and 1 in 70,000 newborns are born with the disease. Each subtype is caused by the alteration of a different enzyme needed to completely break down heparan sulfate. The different types of MPS III have similar signs and symptoms, although type A is the most severe. MPS III is a progressive disorder that primarily affects the brain and spinal cord (central nervous system). Other names for this condition include: MPS III, mucopolysaccharidosis III, and Sanfilippo syndrome.