Biomarin | Lysosomal Acid Lipase Deficiency

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.

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.