Among our members, we have two patient organizations in the cystic fibrosis space, but I'm going to speak about my direct personal knowledge.
I have a son with a rare condition, which is PKU, or phenylketonuria. He is on the first pharmaceutical and doing extremely well with that one. There is a second pharmaceutical for PKU because the first one doesn't work for everybody. The second one was approved in the United States in 2019 and in Europe in 2020. There's no sign of an application in Canada.
The same drug developer is taking that rare disease seriously. They have launched a clinical trial in Europe and in America—but not in Canada—for the first gene therapy for PKU. There's tangible evidence of at least a delay, if not a complete lack of access, to, frankly, breakthrough therapies for the rare disorder. It affects about 2,500 Canadians, but it's one that I'm intimately familiar with. There's no sign that this drug company is going to make an application for a clinical trial for the gene therapy, for the second drug or for the gene therapy, if and when it appears to be safe and effective.
I would be delighted to do a follow-up with the committee with other tangible examples. It's not the only one, but it's the one that's very near and dear to my own heart.