Influencing Policy
We are transforming the landscape, but delivering real change for people living with Duchenne muscular dystrophy depends on effective policy, regulation and commissioning decisions.
Our approach
As a solutions focused organisation, we tackle barriers wherever they exist.
We seek to influence decision makers and system leaders at every level to remove systemic barriers holding back progress and close gaps in care and support for people with Duchenne.
Our policy and advocacy work is wide ranging – from engaging with regulators, providing a voice for people living with Duchenne and their families and representing the community in the medicines approval and reimbursement process with the Medicines Healthcare Products Regulatory Agency (MHRA) and National Institute for Health and Care Excellence (NICE) to leading national campaigns such as Time is Muscle, which helped families secure access to a breakthrough treatment through an Early Access Programme (EAP).
Collaborating to drive change
As members of various coalitions and associations we help shape the health and medicines policy agenda more broadly.
Speaking up for our community
We also speak up for our community on issues that matter deeply to them and work with other charities, industry, disability charities and other groups to call for wider reforms, including improvements to adult social care, welfare, Special Educational Needs and Disabilities (SEND) support, and accessibility within the context of the social model of disability.
We collaborate with people with Duchenne, their families, clinicians, researchers and partners to harness the power of patient advocacy and drive reform where it’s needed most.
Together, we can help build a system that works for rare diseases and delivers meaningful change for those living with Duchenne.
Our campaigns and reports
Harnessing the strength of our community, our #TimeisMuscle campaign called for faster access to a new treatment for Duchenne, givinostat, because every day matters for people living with Duchenne.
Our Transforming our rare reality report sets out clear recommendations for improving diagnosis, speeding up approvals for new treatments, and ensuring fair access to new therapies.
These changes can transform lives—and we’re pushing to make them happen.
