In an editorial written for the web site rarediseaseawareness.ca, Dr. Paul Goodyear, Professor of Pediatrics at McGill University argues that the government should be responsible to ensure that those suffering from disease should have access to the latest and most effective treatments available.
In particular Dr. Goodyear is concerned with those that suffer from rare diseases for which there are no large groups to lobby and act as their voice. He provides the example of cystinosis which is caused by mutations of the gene that provides for free movement of cysteine from one compartment to another within our cells. Children affected with the disease typically require a kidney transplant by the age of 10 and have a life expectancy of about 30 years.
April 2, 2014