Biomarin

04 Jul 2016
Sticky Post By Posted in Advocacy, Morquio, MPS IVA Permalink

Vimizim Compassionate Use Update

Sticky Post By On 4 July, 2016
Some exciting news to share – The Isaac Foundation spent the entire month of June ensuring continued access to Vimizim for our Canadian Clinical Trial patients suffering from Morquio A Syndrome.
 
The compassionate use program, which saw all patients who participated in the Vimizim clinical trial receive access to therapy for a certain period of time, was set to end on June 30, 2016. This would have resulted in the remaining 11 patients losing access to their much needed treatment until it was fully reimbursed by the government.
 
Working collaboratively with the Ministry of Health in Ontario and with Biomarin pharmaceuticals, I’m thrilled to report that we’ve been able to ensure access to treatment for all 11 patients until Biomarin and Canadian Provinces (pCPA) can negotiate terms and conditions for coverage for all patients.  Both Biomarin and the government did fantastic work on this, and we’re thrilled with the outcome.
 
We will continue to do everything we can to help patients and their families receive the best care possible as they battle this and other ultra-rare diseases. Thanks to everyone for supporting us and helping along the way.
 
#Hope, always…
09 Feb 2014
Sticky Post By Posted in Advocacy, MPS IVA Permalink

Morquio A Syndrome (MPS IVA) Advocacy

Sticky Post By On 9 February, 2014

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.