Representing Pompe patients / families of all ages, CAP has members from all across Canada.
The Canadian Association of Pompe (CAP) was incorporated as a non-profit society in 2013, after the initial group of families met in 2006. The Common Drug Review released its recommendation in 2007 that Alglucosidase alpha be listed for patients with infantile-onset Pompe disease. They felt there was insufficient evidence to recommend it be listed for other forms of the disease. Those early years for families were focused on reimbursement for this life-saving enzyme replacement therapy for all patients. It was just in 2014 that the New Brunswick Drugs for Rare Diseases Plan was passed. This was a huge milestone for many people, especially those families directly affected. In 2016, we saw patients in Nova Scotia start treatment. This means that all provinces in Canada that have Pompe patients are treating.
It’s been my pleasure to serve as CAP’s president and now executive director since 2012. I was diagnosed with Pompe disease about twelve years ago so I know very well what some of the challenges are that we all live with every day. It’s not all bad though, I’ve met some wonderful people that I never would have met otherwise.
We’ve seen some significant changes the past few years. It isn’t good to see more patients diagnosed, but it’s good to see them diagnosed with a disease that we know they had anyway. Now they can connect with other patients/families. As we do our best to raise awareness, patients will be diagnosed sooner. CAP has always been a supportive group of families and advocates but, since becoming a non-profit society, we can expand on what we’re able to do.
There’s so much research being done on treatments for rare diseases, including Pompe disease. This is one of very few disorders where a treatment exists, one that works! It won’t be long before there are treatment options and families think less of how this changes their lives, and more on their lives without Pompe disease. These are exciting times.