With families affected by Duchenne muscular dystrophy, Muscular Dystrophy UK has been leading the fight for access to new treatment, Translarna, after NHS England announced that it was delaying its decision on whether to fund and approve the drug.
Translarna, which is already funded in European countries including France, Germany, Italy and Spain, is not available in any part of the UK.
What is Translarna?
Translarna is the first ever effective treatment for Duchenne muscular dystrophy, and one of the first for any muscle-wasting condition, to be approved outside of clinical trial.
The drug was granted conditional approval by the European Commission in August 2014. It is designed to treat boys whose Duchenne is caused by a nonsense mutation, who are aged five and over and who can still walk. Clinical trials of Translarna show that the drug could help keep children walking for longer, and delay the need for a wheelchair.
Why is fast access so important?
Children must still be able to walk in order to obtain the treatment, so months of delays could be the difference between some being able to access the drug or not.
Once lost, muscle fibres cannot be restored. Every day counts.
Get involved and join the fight
Together with families, Muscular Dystrophy UK has taken the fight for access to Translarna to the very top.
We have organised for families to take a massive petition on Translarna to 10 Downing Street. With the support of families and Parliamentary supporters, we have also consistently raised the issue in Parliament and taken the campaign directly to decision makers –NHS England, senior Health Ministers and NICE, even bringing the issue to the attention of the Prime Minister.
We will not let up until Translarna has been funded and approved.
For more information on Translarna and the campaign for access, please get in touch with Peter Sutton on firstname.lastname@example.org or call 020 7803 4838.