Tuesday, December 09, 2008

Sympathy, but no help for a rare disorder

From the Globe and Mail:
Once a husky machinist, Greg Troy is a diminished figure who fights for every breath. He navigates his home in Waterloo, Ont., by grasping pieces of furniture. He can sleep only when attached to a ventilator.

The 62-year-old inherited a rare metabolic disease called Pompe that causes excessive amounts of glycogen to accumulate in the body, disabling skeletal muscles and damaging the heart.

Although there is a drug to treat the often fatal illness - costing up to $600,000 a year - access to it in Canada is unequal. Ontario has a policy to provide it only to babies, where it has been shown to improve survival significantly. Quebec is paying for one infant to receive it, while Alberta funds three patients of various ages under a special grant.

Mr. Troy was refused the enzyme-replacement drug, called Myozyme. In rejecting his request, the Ontario Ministry of Health said in a Sept. 12 letter that there is a lack of evidence concerning the efficacy of the drug in adults - but that it extended its "full sympathy" to Mr. Troy. ...more

No comments: