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

Recent reports of black particles after reconstitution of Myozyme

From Health Canada:
Genzyme has received isolated complaints pertaining to an observation of black gelatinous particles after reconstitution of 50 mg vials of Myozyme® (alglucosidase alfa). Working in close consultation with relevant regulatory authorities, Genzyme immediately initiated an investigation and discovered that when certain types of needles are used to penetrate the rubber stopper on Myozyme vials, black gelatinous particles may appear in the product, on the stopper or on the needle. Although we have not received similar complaints with Genzyme's other products, we are writing to alert our customers to the potential that such particles might appear and to provide current advice and recommendations. ...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