Patient advocacy

In order to ensure new treatments coming down the pipeline can reach Duchenne sufferers as quickly as possible, we believe it is essential to remain at the forefront of the conversation between the policy makers, research institutions, pharmaceutical companies and patients and their families. Any treatment or cure for Duchenne will be fruitless unless patients can access that treatment, and be supported during the treatment process and afterwards. With this in mind, we are continually seeking ways to engage with these groups and push the issue of earlier access to medicine higher up the political agenda.

We are participants in the Newcastle Plan, in which patient organisations, pharmaceutical companies, the National Institute for Health Research (NIHR) and a wide range of NHS clinicians are all working to tackle the challenges of UK clinical trial capacity for Duchenne. The Plan outlines three stages of development over the next five years, intended to cement the UK as a centre for clinical trials for Duchenne muscular dystrophy and ensure all patients with Duchenne – both children and adults – have access to clinical research opportunities. We will remain closely involved in the Plan as part of a patient organisation group that will help co-ordinate the process and identify funding options. To read the full summary document published by TREAT-NMD, please click here.

You can read more about patient advocacy issues at Empower: Access to Medicine.