Rare Disease Report

The Assistance Fund Provides Financial Assistance to Gaucher Patients

MAY 07, 2017
Mathew Shanley
Earlier today, The Assistance Fund announced the opening of a new fund designed to provide financial assistance to patients living with Gaucher Disease.

Gaucher disease, a genetic disorder, occurs in 1 in every 50,000 to 100,000 people in the general population. The inherited condition is the effect of the inability to make glucocerebrosidase, the enzyme responsible for breaking down fatty substances in the body. Consequently, the substances build up in many tissues of the body, including the bones, organs, and bone marrow, and the substances then prevent cells and organs from functioning properly.

Treatment of this chronic condition can be incredibly expensive and, with the new fund, the independent foundation is set to provide eligible patients with financial assistance for copayments, coinsurance, deductibles, health insurance premiums and incidental medical costs.

“Gaucher disease is a rare and complex genetic disease with significant and varied symptoms that often require management by a multidisciplinary team of healthcare professionals,” said The Assistance Fund President, Mark P. McGreevy. “We are pleased to be able to provide financial assistance for adults and children with Gaucher disease with the opening of the broadest fund that we have available, covering copays, insurance premiums and incidental medical expenses for those who qualify for our programs.”

Gaucher disease can be categorized in three different classifications: type 1, type 2, and type 3.

Type 1 is the most common, and involves bone disease, anemia, an enlarged spleen and low platelets (thrombocytopenia). It can affect both children and adults, and is also commonly referred to as non-neuronopathic Gaucher disease because the brain and spinal cord are typically unaffected.

Type 2 is both severe and often fatal. It is known as neuronopathic and begins in infancy with severe neurologic involvement, hypertonia, mental retardation, and apnea. Most children with type 2 Gaucher disease do not live past the age of 2.

Patients with Type 3 Gaucher disease may live into adulthood, as symptoms usually show up later in adolescence. They include liver, spleen, and brain problems, muscle twitches known as myoclonus, convulsions, dementia, and ocular muscle apraxia, osteoporosis, yellowish-brown skin pigmentation.

The Assistance Fund has a history of helping patients and families facing high medical out-of-pocket costs, and its latest fund covers all FDA-approved medications for the treatment of Gaucher disease.

To determine eligibility for assistance, or to learn more, individuals can visit tafcares.org.


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