Mucopolysaccharidosis Type IV
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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 TypeCo-Pay, Co-insurance & Deductible (medications and office visits)
Maximum Award Level$13,000
- 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 IV
Mucopolysaccharidosis type IV (MPS IV), also known as Morquio syndrome, is a rare metabolic condition in which the body is unable to break down long chains of sugar molecules called glycosaminoglycans. As a result, toxic levels of these sugars accumulate in cell structures called lysosomes, these materials remain stored in the body’s cells, causing progressive damage and leading to the various signs and symptoms associated with the condition. Affected people generally develop features of MPS IV between the ages of 1 and 3. These signs and symptoms may include abnormalities of the skeleton, eyes, heart and respiratory system. There are two forms of MPS IV: MPS IVA is caused by changes (mutations) in the GALNS gene and MPS IVB is caused by mutations in the GLB1 gene.
MPS IV is one of the rarest of the MPS diseases in the United States. Reliable incidence figures are not available, but estimates find the disease in 1 in 200,000 to 300,000 individuals. Affected individuals develop various skeletal abnormalities, including short stature, knock knees, and abnormalities of the ribs, chest, spine, hips, and wrists. Other names for this condition include: Morquio-Brailsford disease, Morquio Disease, Morquios Syndrome, MPS IV, and mucopolysaccharidosis (MPS) IV (A,B).
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- Healthwell Foundation800-675-8416
- National Organization for Rare Disorders800-999-6673
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- Patient Services Inc.800-366-7741
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