9 February, 2014 mcfadyena

Morquio A Syndrome (MPS IVA) Advocacy

morquiotitleHi Everyone,

I’m excited to announce that The Isaac Foundation will be working with families throughout Canada to help ensure provincial funding for Enzyme Replacement Therapy (ERT) for MPS IVA patients is forthcoming once the treatment gets approval from Health Canada.

Morquio Syndrome is a rare disease that is part of the MPS umbrella of lysosomal storage disorders.  The disease affects major organ systems in the body and leads to devastating symptoms for sufferers.  These symptoms included bone and joint disease, heart and airway disease, a shortened stature, and premature death.

We have been fortunate to have had success advocating for families seeking support and treatment options for other forms of MPS in numerous provinces over the past 8 years.  Currently, there are no treatment options for sufferers of MPS IVA.  However, recent clinical trials for Biomarin’s Vimizim have produced very promising results, and Health Canada is expected to approve the treatment for use in Canada in the very near future – most likely before the summer of 2014.   A decision by the FDA in the United States is imminent, and an approval in the US could mean that patients can access the treatment via the Federal Government’s Special Access Program.  However, approval through the SAP is always contingent on provincial funding being made available.

We are excited to help advocate with families as they seek treatment options for themselves or their affected children, and will work with the same passion and resolve that we have given all of our other advocacy efforts.

If you or your family is interested in working with The Isaac Foundation to help bring ERT to your province for yourself or your children, please don’t hesitate to contact Andrew McFadyen at HERE or via telephone at 613.328.9136.

For more information about Morquio A Syndrome please click HERE.

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