Meeting with Minister for Life Sciences

The meeting, along with Christine Lavery and Charlotte Roberts from the MPS Society, was attended by the Brown’s, Ms Mallah, whose son headed up the legal challenge against NHS England, and a family whose son is affected with Duchenne Muscular Dystrophy and whose treatment, Translana, has been caught up in this funding decision process delay as well.

The outcome of the meeting was the offer of another meeting this week with MP George Freeman, NHS England and the two pharmaceutical companies whose drugs have been caught up in the delayed decision making process as well. We are hopeful that this will occur on Thursday.

There is so much uncertainty, we cannot assume that the drug will be reimbursed. There is compassionate use for those on the clinical trial, but we don’t know how many more months this will continue. For every week that goes by individuals not on treatment are deteriorating. It has been eight months since the drug was licensed and the waiting began.

Today we should have heard NHS England’s decision regarding Vimizim, however, following the threat of legal action, opposing the scorecard for funding new drugs for rare diseases, it has been scrapped and currently there is no system in place to grant funding for new drugs for rare diseases.

If you would like to write to Prime Minister David Cameron about what Vimizim means to you please follow this link:

Any questions please don’t hesitate to contact me at or 0845 359 9901