You may not have heard much about Fabry disease, but it’s a very serious genetic condition. This disease occurs when a person’s body is unable to make the necessary enzyme required for the metabolizing of fats, lipids, and other related substances.
People with weight problems might think that this disease is blessing in disguise. But Fabry disease is a serious and painful condition, which can severely increase a person’s chances of early death. Plus, without the proper enzymes to break down fat, many serious problems can occur.
First of all, if you suffer from this disease, your body can’t absorb the fat-soluble vitamins A, D, E, and K. These play a variety of roles in your body, such as protecting your eyes, encouraging blood clotting, protecting your bones, and protecting your heart. Also, your body can’t break down these fats in order to flush them out of the body. Instead, they stick around, clogging up your eyes, nervous system, heart, and kidneys.
Because of this buildup, many patients with Fabry disease suffer from stroke, heart attack, and other life-threatening complications. Until recently, there have been few effective treatments for this disease. Anticonvulsants, gastrointestinal helpers, and surgery or dialysis for the kidneys have all been used to help control this disease. But a new drug is showing great promise for sufferers of this rare and painful illness.
The drug is called “Fabrazyme” (agalsidase beta). It was the first drug approved for this condition and it only got acceptance from the FDA in April 2003. It’s a synthetic version of the fat-metabolizing human enzyme and it’s administered intravenously. So, what’s the one problem with this fantastic treatment? It costs around $200, 000 per person, per year.
In the United States, patients have been battling with insurance companies for coverage of this drug. In Canada, the situation has not been very different. Luckily for Canadians, a clinical trial using this drug is now underway, which is open to all residents suffering from this disease. This means that for participants the drug will be covered — at least for the time being.
Hopefully, pressure on both sides of the border will mean that this promising and essential drug will see insurance coverage, so patients with this painful disease can reduce their suffering.