Quebec Morquio A Patient Group Marks first MPS Awareness Day Today, the Communauté Morquio du Québec (CMQ) marked its first International MPS (Mucopolysaccharidosis) Awareness Day by celebrating how far the cause has advanced in the last year, and by calling on all stakeholders in Quebec’s healthcare system to cooperate more closely on a coherent approach to long-term, publicly-funded access to treatment for Morquio A syndrome (MPS IV A).
« It is an honour to lead our new patient group in the fight for equal access to effective treatment options in Quebec for all those affected by Morquio A syndrome, » said Lucie Boudreau, President of the CMQ. This mother of two young men diagnosed with the disease went on to stress that the province’s patchwork approach to treatment access is not equitable, and deprives some patients in Quebec from accessing a drug that they so desperately need.
CMQ spokespersons also took the opportunity on this special day to highlight the importance of their new patient group. « As young Quebecers living with Morquio A syndrome, it has been difficult up to now to connect with others affected by this disease and share advice on how best to manage our health. We have waited a long time for an organization like the CMQ to become established, a group that is dedicated solely to the defense of our cause and which unites us all, » said Laurie Patry-Pelletier, the CMQ’s spokesperson for Eastern Quebec, and Mélissa Bilodeau, the spokesperson for Western Quebec.
An ultra-rare and severely debilitating disease
Morquio A syndrome is a progressive disease that substantially limits the quality and length of life of those affected by it. Unfortunately, there is no cure. Quebecers living with Morquio A face seemingly insurmountable health challenges on a daily basis that impact upon their lung and heart functions, vision, hearing and mobility. Up to now, the only treatment options available to those affected by this disease were multiple surgeries and routine occupational physical therapy. It is estimated that 1 in 200,000 to 300,000 infants are born with Morquio A syndrome. Quebec is understood to have the highest incidence rate in Canada for this disease.
Game-changing treatment options exist, but are still difficult to access
Members of the Morquio A community in Quebec were delighted to learn that Health Canada had approved a new enzyme replacement therapy, VIMIZIM™ (elosulfase alfa), in July 2014. This drug is the first and only available treatment of its kind, and is designed to target the underlying cause of Morquio A and slow its progression. A number of Quebecers living with Morquio A have been treated with VIMIZIM, and have testified to its beneficial, long-term impacts on their quality of life.
The CMQ acknowledges the progress that has taken place since this time last year in securing access to VIMIZIM for a number of Morquio A patients via the Patient d’exception program of the Régie de l’assurance maladie du Québec (RAMQ). However, there are still some patients in Quebec who do not have access to this therapy. Unfortunately, Patient d’exception approvals are on a case-by-case basis and limited to a six-month period. This short timeframe, and the fact that patients must work with their physicians to re-apply every six months, raises uncertainties over sustained coverage for patients in need. A long-term funding solution for VIMIZIM is needed so that those who are affected by Morquio A in Quebec have access to this effective new treatment.
« Quebec’s Morquio A community can rely on the CMQ to promote its interests now, and in the future, » said Lucie Boudreau. « Our message today is clear: we are here for you. Together, we can make a difference and beat this debilitating disease. »
Established in early 2016, the Communauté Morquio du Québec is the province’s first and only dedicated patient support group for those affected by Morquio A syndrome, a rare and severely debilitating disease. The CMQ’s aim is to raise awareness of Morquio A, to support individuals and families affected by this disease, and to advocate for equal and long-term access to new and effective treatments. For further information, please visit www.morquioquebec.org.
