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Fabry Disease

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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

$2,600 Per Year

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 Fabry Disease

Fabry disease is a rare inherited disorder of fat metabolism caused by a defective gene (GLA) in the body, and belongs to a group of diseases known as lysosomal storage disorders. In most cases, the defect in the GLA gene causes a deficient quantity of the enzyme alpha-galactosidase A (Gb3) which is needed to breakdown lipids (fatty substances) in the body. When proper metabolism of this lipid and other similar lipids does not occur, Gb3 accumulates in the majority of cells throughout the body. The resulting progressive lipid accumulation leads to damage and organ dysfunction in the body’s cells, particularly affecting small blood vessels, the heart and kidneys. The cell damage causes a wide range of mild to severe symptoms including progressive kidney damage, heart attacks and strokes often at a relatively early age. Fabry disease can affect males and females of all ethnic and cultural backgrounds.

There are two major disease phenotypes: type 1 “classic” and type 2 “later onset” subtypes. Type 1 occurs in 1 in 40,000 to 60,000 males, with symptoms beginning in childhood or adolescence. Symptoms increase with age, primarily due to progressive glycolipid accumulation in the micro-vascular system. Type 2, or later onset, is more frequent occurring in I in 1,500 to 4,000 males. Classic symptoms of Fabry disease include a dark-red, spotted skin rash in the trunk (angiokeratoma), reduced ability to sweat (hypohidrosis), burning or hot pain in the hands and feet triggered by stress, fatigue or fever (acroparesthesias), and clouding of the corneas.

Other names for this condition: alpha-galactosidase A deficiency, Anderson-Fabry disease, angiokeratoma corporis diffusum, angiokeratoma diffuse, ceramide trihexosidase deficiency, GLA deficiency, hereditary dystopic lipidosis and lysomal storage disorder.

Fabry 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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