Patient Organisations play a pivotal role in helping regulators and policy makers understand the realities of living with Duchenne muscular dystrophy. We need to engage with them early in the drug development process to ensure that when drugs do become appoved for use, the NHS can afford to pay for them, and understands the true value of these potential treatments for patients and families living with DMD.

Duchenne UK engages on a number of issues, from how to speed up the drug development process, to advising on the design of clincial trials and advising on what outcomes are meaningful for patients when drugs are being developed. We work at UK level with the MHRA, and both Alex and Emily are members of the MHRA's Patient Consultative Forum.

The Campaign For Early Access To Medicines


We’ve lobbied with the Empower campaign, which has close links to the Minister for Life Sciences, George Freeman MP, for earlier access to medicines.

We’re pressing the Medicines and Healthcare products Regulatory Agency in the UK, MHRA, to update their Early Access to Medicines scheme - so that companies can seek Conditional or Marketing Authorisation after a Phase II, not after a lengthy costly Phase III.

Picture: Emily Crossley and Alex Johnson, co-founders, Duchenne UK, with the Minister for Life Sciences George Freeman MP, and the Rt Hon Earl Howe, formerly Health Minister

European Medicines Agency (EMA) 

It is important to engage with the regulatory process at European level, and Alex was involved in an exon skipping workshop to engage all stakeholders at the EMA. A paper will shortly be published on the outcomes of the meeting.

Accelerated Access Review

The government is currently undertaking the Accelerated Access Review, which is looking at ways to speed up access to innovative drugs, devices and diagnostics for NHS patients.

Emily and Alex made contributions on the views of patients at various workshops at the MHRA and at a series of discussions organised by the Centre for the Advancement of Sustainable Medical Innovation (CASMI), whose reports fed back to the Accelerated Access Review.

Feedback on the Interim report, was published in January 2016. 

Empower: Data4Health

Duchenne UK attended the launch of the Empower: Data4Health campaign. The campaign is calling for a central database of health records that empowers patients, doctors and researchers, and helps to deliver better treatment across the country. Life Science Minister George Freeman said he looked forward to being hounded by Empower: Data4Health, who’s mission it is to make this happen, because a modernised, digitised health system is the only way forward.

Graham Silk, leukaemia survivor and co-founder of Empower: Data4Health knows this first hand:  

I was diagnosed with leukaemia in 2001, and I’m still here today because of medical research facilitated by the patient data of the leukaemia community...I want everyone to benefit in the way I have.

Food & Drug Administration’s (FDA) Open Public Hearing

Emily and Alex were among 42 parents who travelled to the US in April 2016, to witness the Adcomm for Eteplirsen, an exon skipping drug being developed by Sarepta Therapeutics. Read more about this in our news section.


Glossary of organisations and what they do: 

MHRAThe Medicines and Healthcare Products Regulatory Agency regulates medicines, medical devices and blood components for transfusion in the UK.

EMA: The European Medicines Agency is a European Union agency responsible for the protection of public and animal health through the scientific evaluation and supervision of medicines.

FDAThe Food and Drug Administration is the US body that regulates drugs and advances public health.

NICE: National Institue for Health and Care Excellence is a UK public body which advises on quality standards and information services for health, public health and social care. NICE assesses new drugs to see if they are safe to be prescribed.

NHS England: National Health Service England is the body that leads the National Health Service in England. They set the priorities and direction of the NHS and commission health care services in England.