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Pompe Disease

Co-Pay Relief Program Fund Notices

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Status
Identified

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

Maximum Award Level

$5,900

Eligibility Requirements
  • 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 Pompe Disease

Pompe disease is a rare multisystem inherited disorder caused by mutations in the gene that makes an enzyme called acid alpha-glucosidase (GAA). The body uses GAA to break down glycogen (a complex sugar), without the proper function of this enzyme, the glycogen cannot be degraded to a simple sugar like glucose. In Pompe disease, mutations in the GAA gene reduce or completely eliminate this essential enzyme. Excessive amounts of lysosomal glycogen accumulate everywhere in the body, but causes the most damage to tissue structure and functioning in skeletal muscle, smooth muscle and cardiac muscle. Researchers have identified up to 300 different mutations in the GAA gene that cause the symptoms of Pompe disease, the severity of the disease and the age of onset are related to the degree of enzyme deficiency. Pompe disease may also be classified as a glycogen storage disease, a group of metabolic disorders characterized by abnormalities involving the use and/or storage of glycogen.

Researchers have described three types of Pompe disease, which differ in severity and the age at which they appear. The classic form or infantile-onset Pompe disease usually presents during early infancy (4-8 months of age); The non-classic form or infantile-onset Pompe disease usually appears by age 1 or 2; The late/later-onset type of Pompe disease may not become apparent until after age 2, adolescence, or adulthood. In general, the later the age of onset, the slower the progression of the disease. It is estimated that Pompe disease occurs in 1 in every 40,000 births. Other names for this condition: acid maltase deficiency (AMD), alpha-1,4-glucosidase deficiency, acid alpha-glucosidase (GAA) deficiency, glycogen storage disease type II (Type II GSD), glycogenosis Type II, GSD II, and GSD2.

Pompe Disease Resources
Medication & Financial Assistance Resources
The Patient Advocate Foundation's (PAF) Co-Pay Relief (CPR) Program does not review the information contained on the website links provided for content, accuracy or completeness. Use of and access to this information is subject to the terms, limitations and conditions as outlined on the accessed websites. PAF Co-Pay Relief Program makes no representation as to the accuracy or any other aspect of the information contained on any website accessed from the CPR website, nor does PAF Co-Pay Relief Program necessarily endorse the website information provided. The information presented on the PAF Co-Pay Relief website is provided for general information only and is not intended as a substitute for medical care. Please talk with your healthcare provider about any information you acquire from this or any other website accessed through the PAF Co-Pay Relief program website.
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