In recent years, there has been great progress towards new treatments for some muscle wasting conditions.
We’ve helped to make sure that people can access 10 treatments for five different muscle wasting conditions.
But this is just the beginning. We’re campaigning for access to new treatments and a system that’s ready for the treatments that come next. We won’t stop here.
It moves slowly, relies on processes that don’t work well for rare conditions, and often doesn’t take full account of what life with a rare condition is really like.
That’s why we’re exploring what needs to change and speaking to other organisations facing similar challenges. Together, we will make the system fairer and faster for rare conditions.
Changing the system will take time. In the meantime, we’re doing everything we can to help the treatments currently being assessed get approved.
Treatments that have a lot of evidence about their benefits are the ones that are most likely to be approved. That’s why it’s so important that we show the impact that treatments can have – and make sure everyone involved in the process understands the reality of living with a muscle wasting condition.
If you’ve received any of the treatments below, we’d love to hear your experience.
