canada | Lysosomal Acid Lipase Deficiency

11 Feb 2015

Sticky Post By mcfadyena Posted in Blog Postings Permalink

Petition – Quebec Funding for Vimizim

Sticky Post By mcfadyena On February 11, 2015

Hi Everyone,

Sorry for the delay in posting, I’ve been on the road non-stop meeting with patients and families, and working hard to ensure that reimbursement for treatments for rare diseases is forthcoming for everyone that needs it through this country.

I wanted to share a petition, with hopes you can drop in to sign. It only takes a moment, and the voice you add in that moment can make an incredible difference.

This is a french petition, asking the government of Quebec to fund access to Vimizim for patients with MPS IV. Below is a rough translation for English speaking visitors:

Why it matters

VIMIZIM is a drug that already allows multiple people with Morquio syndrome to live a full life with dignity. INESS has refused reimbursement of the drug, which is too expensive for people to access without help. In Quebec, there are many families in need of this treatment in order to participate fully in society. Furthermore, administered at a young age , this treatment can drastically change the life of a child, or even save his life.

Please visit and sign the petition here!

Thanks in advance,

A.

14 Aug 2014

Sticky Post By mcfadyena Posted in Blog Postings Permalink

New Website Launched!

Sticky Post By mcfadyena On August 14, 2014

It’s official – we’ve launched!

With the approval of VIMIZIM in Canada, the first enzyme replacement therapy available for Morquio A syndrome, we’ve received many questions from across the country. Most prevalent among them is “What’s next?”

After speaking with many families, we thought it would be helpful to put together a website with as much information as possible about Morquio A syndrome in Canada, including information about gaining access to treatment throughout the country.

As many of you know, VIMIZIM was approved by Health Canada in early July, 2014. That approval provided hope for families that they could finally access a treatment for their disease. Clinical trial results are incredibly promising, and patients need to have access to that therapy as soon as possible.

Vimizim Approved

Sticky Post By mcfadyena On July 7, 2014

HEALTH CANADA APPROVES VIMIZIM TO TREAT RARE DISEASE

First Ever Treatment For Morquio A Syndrome; Advocacy Group Calls For Expedited Access For Patients Throughout Canada

CANADA (July 7, 2014) – Health Canada has announced approval of Vimizim, a new enzyme replacement therapy (ERT) to treat Morquio A syndrome, a rare, devastating, and progressive disease that affects 70-100 people in Canada. Vimizim is manufactured by Biomarin Pharmaceuticals (BMRN) and is the first ever treatment available for this rare disorder.