President Guy Ashford-Smith

Vice President Ian McPherson

Secretary Linda Pare

Treasurer Koreen Skelton

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Disease severity in children and adults with Pompe disease varies and is related to age and disease duration, M.L.C. Hagemans,Neurology 2005;64:2139–2141

Canadian Association of Pompe

Founded in Toronto in November 2006 by a group of Pompe families, patients and care-givers, the Canadian Association of Pompe (CAP) is set up to educate Canadians about Pompe disease and to support those diagnosed with the devastating disease. With members from across the country, the organization aims to represent and be helpful to people from each province.

Pompe disease, also known as Acid Maltase Deficiency or Glycogen Storage Disease Type II, is an extremely rare and often fatal neuromuscular disorder caused by a deficiency in the enzyme Acid Alpha-glucosidase (GAA) which serves to break down glycogen, a sugar used for energy. In patients with a deficient GAA enzyme, the glycogen is not broken down and builds up causing progressive weakness in muscles throughout the body particularly the heart, skeletal muscles, liver and nervous system. In babies, the most severely affected, the prognosis is death within one year. If diagnosed as a juvenile, life expectancy is two to three decades. Adults diagnosed with the disease become wheelchair dependent and often require a respirator to breathe. It is a very difficult life for anyone. A deadly disease that takes a devastating toll on those diagnosed and their

families, Pompe affects fewer than 10,000 people worldwide and as many as 30 or as few as 15 in Canada . For the first time, the Pompe

community now has reason to hope for better lives for themselves or their loved ones. Health Canada recently approved a treatment called

Myozyme. It is a costly therapy. However, with so few patients in Canada , the impact on provincial budgets will be minimal. With access to this

treatment, a painful death can be avoided and parents can watch their children grow up and, hopefully thrive. Pompe is a progressive disease. As

each day passes, patients get sicker. It is not known if Myozyme will reverse damage already done. Our goal is to get access to Myozyme early

after diagnosis in order to halt the progression of this disease. We urgently ask for your support in raising this matter with the ministers of health in

each province. Please post this letter to your website and copy it and send it to as many people as you know. For more information about the CAP

and if you would like to know who to talk to in your province, please go to www.pompecanada.com. Thank you for your support.