vimizim | 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.

19 Nov 2014

Sticky Post By mcfadyena Posted in Blog Postings Permalink

BREAKING NEWS! TREATMENT APPROVED IN ONTARIO!

Sticky Post By mcfadyena On November 19, 2014

TREATMENT APPROVED! ONTARIO CHILD FIRST TO RECEIVE PROVINCIAL FUNDING/REIMBURSEMENT FOR VIMIZIM!

We have some very exciting news to share. A few months ago, we told you about little Ayub and our quest to have his treatment approved by the Ontario Government.

I’m thrilled to announce that Ayub will be the first person in Canada to receive full reimbursement for VIMIZIM, a new therapy approved by Health Canada in July and the first and only front-line treatment for Morquio A Syndrome (MPS IVA). This will greatly impact Ayub’s quality of life, and I’m proud to have played a small role in ensuring approval was forthcoming for him.

I’ll post some updates once treatment begins, and will continue working and advocating for the rest of the patients across Canada who need access to this therapy.

11 Sep 2014

Sticky Post By mcfadyena Posted in Blog Postings Permalink

New Diagnosis in Ontario

Sticky Post By mcfadyena On September 11, 2014

This beautiful little boy, Ayub (almost 3 years-old) was just diagnosed with MPS IVA (Morquio A Syndrome) today in Ontario. There is #HOPE for him because the treatment he needs has been approved by Health Canada. The only thing preventing him from beginning treatment ASAP is, once again, the issue of Provincial Reimbursement by the Ontario government.

We will be working hard to help Ontario gather the information needed so they can make the right decision for Ayub and his family, and we will do whatever we can to ensure that treatment begins ASAP!

16 Aug 2014

Sticky Post By mcfadyena Posted in Blog Postings Permalink

Meeting With Heather Forsyth

Sticky Post By mcfadyena On August 16, 2014

Just a quick update…

I’m in Calgary meeting with Wildrose MLA Heather Forsyth. Heather is the official opposition Health Critic, and one of (of not THE) best Health Critic in Canadian politics. Heather works to do what’s best for Albertans, and has advocated for our kids suffering from rare diseases for a long time.

Last year, Heather was instrumental to ensuring that Aleena Sadownyk, a little girl from Alberta, received funding for her enzyme replacement therapy (ERT). Without Heather, I truly believe that Aleena would still be fighting for treatment today.

Heather and I have met numerous times regarding Morquio A Syndrome and the recently approved treatment (ERT through VIMIZIM). She believes, like us, that reimbursement for this treatment should be approved immediately.

In Alberta, there are 5 cases of Morquio A Syndrome. With Heather’s help, it is my hope that all of these patients requiring treatment can begin as soon as possible.

Heather and I will stay in touch, and she’ll continue to play a big role in advocating for our patients. I’m grateful to have her support, and I’m proud to call her a friend.

I’m heading to Edmonton on Monday to meet with PC MLA Stephen Kahn and the head of Pharmaceutical Funding and Guidance in Alberta. I’ll update more as soon as I can.

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.

Drawing Hope – Morquio A

Sticky Post By mcfadyena On May 28, 2014

What a great project!

Luke and Zane Braun, two boys from Ontario battling Morquio A Syndrome, were featured by photographer Shawn Van Daele for his DRAWING HOPE PROJECT.

Luke and Zane were asked to complete a drawing showing what life would be like for them after receiving ERT treatment with VIMIZIM. Shawn then takes that drawing, does a photo shoot with the boys, and hits his home studio to edit his subjects into their “dream”. The results are stunning! See the images below and visit Shawn’s page over at www.drawinghope.ca.

Luke, Zane, and Shawn’s journey with the DRAWING HOPE PROJECT was featured on CTV news as well!