A joint statement from Emily Crossley of Duchenne Children’s Trust and Alex Johnson of Joining Jack:

Today we attended a tense, highly charged and ultimately frustrating meeting with George Freeman MP to discuss #‎translarna. The meeting was organised by Greg Mulholland MP who is doing a brilliant job for our children. Alex Johnson made the journey from Wigan early this morning to attend the meeting. But in a disgraceful no show, @NHSEngland, whose offices are based around the corner from The Department of Health, did not send a representative - despite having given assurances that they would. This is the second time they have not turned up at a meeting to discuss Translarna. Our children's lives are in their hands, and yet they do not turn up to to a meeting with the Minister for Life Science. Our meeting focused on 3 drugs currently under review for possible funding by NHS England: A drug for Morquio disease, another for Tuberose sclerosis complex and Translarna a drug to treat 13% of boys with DMD. While NHS England have been dragging their feet in making a decision on these drugs, one Morquio patient has died. This is really appalling and should not be allowed to happen again. 

Here are the questions we would have liked to have asked NHS England if they had bothered to show up.

1) What is the methodology behind your decision making process on potentially life-saving drugs for children who are dying? Why won't you share it with us? 

2) Why will you not give us a date for a decision on whether you will fund these drugs? Your consultation ends on April 27th. We need to have a date for a final decision. In the case of Tuberose Sclerosis, you have been consulting for over 2 years. It's unacceptable. 

3) Why are you misleading the government that you are in discussions with the pharmaceutical companies about extending access to drugs, when the pharmaceutical companies tell us that you are not actually in discussion with them at all? 

We now need answers. As Professor Kate Bushby told the APPG on Muscular Dystrophy, “NHS England's handling of the process of approving rare disease drugs like Translarna…has been shambolic” 

We hope that George Freeman will honour his promise to promised to look into this - before parliamentary recess on Monday. Please don't let NHS England get away with this.

Alex and Emily