Current Campaigns: Our Fight for Vimizim

Current Campaigns: Our Fight for Vimizim

Over the last five years 34 MPSIVA patients have been part of a clinical trial for a treatment in the UK. This involved weekly travel to hospital for four hours of infusion. Hospitals were often hundreds of miles of home either resulting in a 12 hour day or an overnight stay in a nearby hotel. While the UK led on the clinical trial yet we are one of the last countries in Europe to have funding approved. The treatment Vimizim was licensed in April 2014 and over 20 countries have since agreed to fund the treatment.

Those who were part of the clinical trial are still compassionately receiving the treatment but it is uncertain how much longer this will continue. Unfortunately those were not involved in the clinical trial are being denied treatment until funding is approved.

A funding decision was expected mid-December but NHS England failed to develop a suitable decision making process for ultra-rare disease. Through supporting one of our ten year old members with Morquio to legally challenge the scorecard system, NHS England are now redeveloping their processes.

For all the news concerning our fight for Vimizim, please take a look at our 'Vimizim: news and information' page.

This decision is bittersweet as the ongoing delay means that a number of members who would benefit from treatment are denied access until a funding decision is made whilst others are continuing to travel hundreds of miles weekly to hospital for treatment which could be received at home.

So why is Vimizim so important? Take a look at some statements from families and individuals affected by MPSIVA to learn more.

Please meet some of our members by clicking on their names to learn what Vimizim means to them.

Sam

“Vimizim is changing Sam’s life. This is clinically proven, but I don’t need clinical evidence. I can see it every day.”

Kamal

“Kamal’s only wish for Father Christmas is ‘to be a normal boy’, and by having Vimizim it gives him the chance to be as normal as can be.”

Angela

“…just being able to get on with life and enjoy my leisure time as everyone else does, without constantly feeling exhausted, has had a very big impact on my quality of life.”

Gracie

“We can’t even bear to think about the fact that it may not be funded. It would feel like we had given Gracie the best chance, to have it snatched away from her.”

Sarah

”..I have noticed my body really craves the enzyme replacement treatment, I’m very scared about it discontinuing and going back to that dark place.”

Lily-May

“All of us deserve a chance. All of us deserve a feeling of normality. I am a fighter, because I have no choice and I have everything to fight for.”

Harvey

“We are extremely angry that our baby’s life is not important enough for the treatment to become available to him. We are losing faith in our country and health system”

This phenomenal testimony has been written by a young woman who spent six months off Vimizim. Please read her story here.

It has only been a six months since I’ve been put back on treatment, and I already feel a difference. I notice I am able to do new things every day, and am meeting many physical milestones which would have been impossible without having taken Vimizim again.”