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 clinical 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.
Information on important organisations and regulators.Read more
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.Read more
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.Read more
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.Read more
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.Read more