Group calls for national policy to cover drugs for rare diseases

From CBC News:
A group that represents Canadians with rare disorders is calling for a national policy to help pay for their astronomical medical costs.

The call comes after the Alberta government decided to pay for a drug for a 17-year-old boy stricken with Pompe disease, a rare and potentially fatal disease caused by an enzyme deficiency that disables muscles.

Trevor Pare of Innisfail has been a participant in a clinical trial that paid for the drug Myozyme. The trial is over at the end of May and the Alberta government has decided to take over the annual cost, which is more than $500,000.

"It's just like unbelievable, unbelievable," Linda Pare, Trevor's mother, told CBC News. She said without the drug, her son would die in six months.

The Canadian Organization for Rare Disorders is pleased Trevor will continue to get his special drug.

But Durhane Wong-Rieger, president of the organization, said it is time for a national policy on funding of drugs used to treat rare diseases. ...more

Teen fears he may die without test drug

From the Globe and Mail:
An Alberta teenager fears he could die within six months if he cannot get an experimental drug for an extremely rare disease.

Seventeen-year-old Trevor Pare suffers from Pompe Disease and has been taking the drug Myozyme for the past four years as part of a clinical trial.

But the trial runs out at the end of May, leaving his family on the hook for the nearly $500,000 annual cost.

Pompe is a hereditary disease caused by a mutation in the gene that produces the enzyme alpha-glucosidase, which the body needs to break down glycogen. ...more

Experimental drug is Ian's only hope

From the Hamilton Spectator:
Hope was the last thing Ian MacPherson expected.

Pompe Disease was marching through his body on schedule, disabling his muscles and lungs. There was nothing doctors could do to stop it.

"I didn't expect any treatment in my lifetime," he said.

But then his parents discovered Myozyme on the Internet. The drug was so new it was still being tested in rabbits. Desperate, they requested it anyway. They were denied. It was too experimental and expensive. ...more